A gathering of scientists might not seem very glamorous, but the cancer researchers who came together at the Hyatt Regency Greenwich Thursday evening were celebrities at a gala marking the 10th anniversary of a local nonprofit that has provided more than $22.5 million to help investigate innovative therapies.

The Alliance for Cancer Gene Therapy celebrated its founding 10 years ago by Greenwich residents Barbara and Edward Netter, who decided to raise money for research into gene therapy after losing their daughter-in-law to breast cancer.

The event Thursday served as a tribute to Edward, who died last February. It also allowed the scientists to gather and discuss the cutting-edge research they are doing, and how the Alliance can help along the often painstakingly slow process of bringing new therapies to the market.

A few years ago, during the event celebrating the fifth anniversary of the Alliance, the scientists' discussion led to the eventual awarding of grants that recently paid off with a major breakthrough.

The nonprofit had already been funding so-called young investigators conducting exploratory research.

"We also envisioned there was a lack of science and clinical development," said Dr. Savio Woo, chairman of the Alliance's scientific advisory council. "You need to test patients."

Since then, the Alliance has begun giving out its Fund for Advancement awards, which provide up to $1 million to scientists conducting research that will move cell and gene therapies from the laboratory toward clinical trials.

Last year, an Alliance-funded gene therapy trial at the University of Pennsylvania caused two patients with chronic lymphocytic leukemia to go into complete remission, and significantly reduced the cancer in a third patient.

Dr. Carl June, who headed the small trial, which genetically modified the patients' own T-cells to target and kill the tumors, called the gathering Thursday a "celebration" of the work the Alliance has been supporting.

"After a decade, it's clear there are going to be FDA-approved products in cell and gene therapy that there weren't 10 years ago," June said.

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Greenwich gala celebrates cancer therapy breakthrough

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18-04-2012 06:57 Many of our patients travel to Guangzhou from all over the world for medical treatment and tourism. China medical tourism can help with becoming a patient, travel arrangements and language assistance. If you want to know more about our services, please browse the web: or mail to us: Name: Sonia Tahiliani Gender: Female Nationality: United Kingdom Age: 10 Start of treatment: 2007-08-02 Diagnosis: Cerebral palsy quadriplegic with epilepsy Treatment: Stem cell treatment Days admitted to the hospital: 120 Reason for coming to treatment: Sonia was a ten-year old girl, and she was born with Cerebral Palsy. She is quadriplegic, suffers from epilepsy and delayed physical and cognitive development. She cannot walk or speak; she can make a few simple sounds and can minimally respond to commands. She has great difficulty remembering and learning new things. Her parents were hoping their girl to be as healthy as a normal kid, so they took her here out of trust in our medical technology.

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China medical tourism Cerebral Palsy stem cells therapy 10 - Video

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18-04-2012 09:35 Video update that includes Holly Catalano crawling after her second stem cell treatment at the Stem Cell Institute in Panama. At the end of the video is additional footage showing Holly walking along furniture after her third treatment. Holly suffers from periventricular leukomalacia, a disorder which is similar to CP.

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Stem Cell Treatment for Cerebral Palsy - 3rd Treatment Update Holly Catalano - Video

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BOSTON--(BUSINESS WIRE)--

Inform Genomics, Inc., a private company focused on developing novel platforms of personalized medicine products for cancer supportive care and inflammatory diseases, today announced the completion of the first phase of product development to predict a patients risk of developing oral mucositis after receiving high dose chemotherapy prior to hematopoietic stem cell transplant. The results of this single center, 153-patient study demonstrated the products ability to discriminate which patients develop oral mucositis with 99.3% accuracy and an area under the Receiver Operator Characteristic (ROC) curve of 99.7%. Further development will include validation of these initial results in a multicenter study. In addition, Inform Genomics announced that it entered into a collaboration agreement with Swedish Orphan Biovitrum AB (Sobi) to further develop and commercialize the product. Sobi is a leading integrated biopharmaceutical company dedicated to bringing innovative therapies and services to improve the health of rare disease patients and their families.

We are very pleased with the exciting results of this study, said Ed Rubenstein, M.D., President & CEO of Inform Genomics, and our agreement with Sobi demonstrates the value our technology can bring to biopharma partners while expanding the market opportunity for both companies products. When commercialized, this product will be available for the hematology oncology stem cell transplant market and will complement the target market of our lead product, OnPART for patients with solid tumors.

The principal investigator for the study, Stephen T. Sonis, D.M.D., D.M.Sc., Chief Scientific Officer of Biomodels, LLC, who also serves as the Chief of the Division of Oral Medicine at the Dana-Farber Cancer Institute and Professor of Oral Medicine at the Harvard School of Dental Medicine, will present the results of the study at the upcoming 2012 American Society of Clinical Oncology (ASCO) Annual Meeting, as part of the educational session titled Mucosal Injury in Patients with Cancer: Targeting the Biology, taking place from 11:30 am to 12:45 pm on Sunday, June 3, 2012 in Chicago, IL.

About OnPART

OnPART, Oncology Preferences And Risk of Toxicity, will be Inform Genomics first platform molecular diagnostic test for personalizing treatment decisions for patients undergoing chemotherapy for colorectal, breast, lung or ovarian cancer. Based upon response rates and survival, more than one chemotherapy regimen may be considered appropriate care for patients with these common solid tumors, yet the regimens vary widely in their toxicity profiles, including nausea & vomiting, diarrhea, oral mucositis, cognitive dysfunction, fatigue and peripheral neuropathy. OnPART is being developed to assess genomic risk for these side effects, and to provide valuable information for patients and medical oncologists to help clarify clinical choices.

About Inform Genomics

Inform Genomics, Inc. is a private company focused on developing novel platforms of personalized medicine products for cancer supportive care and inflammatory diseases, including its lead product, OnPART, designed to predict an individuals risk of six common toxicities of commonly used chemotherapy regimens based on his or her individual genomic profile. The Companys business model leverages existing technology in conjunction with proprietary analytic methods for conducting genome-wide association studies. Product development programs will lead to commercial, single source laboratory tests consisting of single-nucleotide polymorphism (SNP) clusters that determine the likelihood of individual patient clinical outcomes to drug therapies. The U.S. market opportunity for these differentiated products exceeds $2 billion annually. Inform Genomics is headquartered in Boston, Massachusetts. For more information, please visit http://www.informgenomics.com.

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GRAND RAPIDS, Mich., April 19, 2012 /PRNewswire/ --Researchers at Lund University in Sweden have discovered a new stem cell in the adult brain. These cells can proliferate and form several different cell types -- most importantly, they can form new brain cells. Scientists hope to take advantage of the finding to develop methods to heal and repair disease and injury in the brain.

Analyzing brain tissue from biopsies, the researchers for the first time found stem cells located around small blood vessels in the brain. The cell's specific function is still unclear, but its plastic properties suggest great potential.

"A similar cell type has been identified in several other organs where it can promote regeneration of muscle, bone, cartilage and adipose tissue," said Patrik Brundin, M.D., Ph.D., Jay Van Andel Endowed Chair in Parkinson's Research at Van Andel Research Institute (VARI), Head of the Neuronal Survival Unit at Lund University and senior author of the study.

In other organs, researchers have shown clear evidence that these types of cells contribute to repair and wound healing. Scientists suggest that the curative properties may also apply to the brain. The next step is to try to control and enhance stem cell self-healing properties with the aim of carrying out targeted therapies to a specific area of the brain.

"Our findings show that the cell capacity is much larger than we originally thought, and that these cells are very versatile," said Gesine Paul-Visse, Ph.D., Associate Professor of Neuroscience at Lund University and the study's primary author. "Most interesting is their ability to form neuronal cells, but they can also be developed for other cell types. The results contribute to better understanding of how brain cell plasticity works and opens up new opportunities to exploit these very features."

The study, published in the journal PLoS ONE, is of interest to a broad spectrum of brain research. Future possible therapeutic targets range from neurodegenerative diseases to stroke.

"We hope that our findings may lead to a new and better understanding of the brain's own repair mechanisms," said Dr. Paul-Visse. "Ultimately the goal is to strengthen these mechanisms and develop new treatments that can repair the diseased brain."

Link to the study here:

http://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0035577

About the Neuronal Survival Unit, Faculty of Medicine, Lund University

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New Stem Cell Found in the Brain

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SEATTLE and SINGAPORE, April 19, 2012 /PRNewswire/ -- Cell Therapeutics, Inc.("CTI") (Nasdaq and MTA: CTIC) and S*BIO Pte Ltd announced today that the companies have entered into an asset purchase agreement pursuant to which CTI would acquire world-wide rights to S*BIO's pacritinib, a highly selective JAK2 inhibitor. Pacritinib is an oral JAK2 (Janus Associated Kinase 2) selective inhibitor that has demonstrated encouraging clinical benefit in phase 1 and 2 clinical studies in patients with primary myelofibrosis (MF) or MF secondary to other myeloproliferative neoplasms (MPN). Pacritinib has orphan drug designation in the U.S. and Europe for myelofibrosis.

"JAK2 dysregulation is associated with a broad range of difficult-to-treat illnesses, including cancers and autoimmune diseases, and is one of the most exciting potential new targets in cancer therapy today," said James A. Bianco, M.D., CEO of Cell Therapeutics, Inc. "We believe a highly selective JAK2 inhibitor that also inhibits the JAK2 clonal mutation (JAK2V617F) offers a distinct biological and clinical advantage over marketed or development stage compounds which are non-selective inhibitors of the JAK pathway. We believe that the lack of suppression of red blood cell and platelet formation seen with pacritinib has the potential to satisfy a medical need not currently addressed with existing non-selective JAK1/JAK2 inhibitors."

"The acquisition of pacritinib is aligned with our strategy of becoming a leader in the treatment of blood related cancers and disorders. We are looking forward to build on the progress made by S*BIO," Bianco continued. "With Pixuvri approaching approval and launch in the EU, and tosedostat and pacritinib entering phase 3 our late stage portfolio addresses a full complement of blood related cancers ranging from MPN to MDS, leukemia and lymphoma."

"JAK inhibitors are a very exciting new class of targeted agents that provide effective treatment in a previously difficult to treat disease called myelofibrosis," stated Srdan Verstovsek, M.D., Ph.D., Associate Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas and Executive Committee Member, International Working Group for Myelofibrosis. "Pacritinib is a highly specific JAK2 inhibitor that does not appear to cause suppression of platelets or red blood cells as seen with other treatments, while reducing enlarged spleen and improving disease related debilitating symptoms in patients with myelofibrosis. I am looking forward to working with Cell Therapeutics in developing this agent for patients with myelofibrosis who present with low blood counts or develop them on therapy."

"We are most impressed with CTI's experienced team and believe that they are the ideal company to further develop pacritinib, S*BIO's most advanced program," commented Ms. Tamar Howson, S*BIO's CEO. S*BIO is a privately-held biotechnology company focused on the research and clinical development of novel targeted small molecule drugs for the treatment of cancer with leading programs around kinases and histone deacetylases (HDAC). S*BIO has strong links with a network of medical oncologists in Asia Pacific and its investors include Bio*One Capital a subsidiary of EDBI, Aravis Ventures, Mitsui Ventures and other international funds.

Pursuant to the terms of the agreement, CTI will make an upfront payment of $15 million and issue $15 million shares of unregistered preferred stock convertible into common stock in CTI. The agreement also includes regulatory success- and sales-based milestone payments, as well as single digit royalties on net sales. CTI will be solely responsible for development and commercialization activities of pacritinib worldwide. The agreement will be subject to satisfaction of certain closing conditions. The terms of the agreement will be provided in more detail in a Form 8-K to be filed with the U.S. Securities and Exchange Commission.

About Janus Associated Kinase (JAK)

The JAK family of enzymes are a central component in signal transduction pathways, which are critical to normal blood cell growth and development as well as inflammatory cytokine expression and immune responses. When dysregulated by activating mutations, uncontrolled blood cell growth can occur accompanied by inflammation and immune system activation contributing to disease manifestations in MPN. Autoimmune diseases such as psoriasis and rheumatoid arthritis also have activation of this pathway and JAK inhibitors are in development for these disorders. In addition, activation of the JAK2 pathway and the related FLT3 pathway (whether by activating mutations or other causes) is frequently associated with leukemia, and lymphoma. Pacritinib inhibits both JAK2 and FLT3 suggesting potential use in treating such blood related cancers.

Myelofibrosis is a stem cell-derived clonal myeloproliferative disease frequently associated with a mutation in the JAK2 gene (JAK2V617F). Inhibition of JAK1/ JAK2 has recently been shown to lead to clinical benefit in patients with advanced MF and platelet counts of 100,000 or higher at study entry, resulting in the first JAK1/JAK2 inhibitor to be approved for patients with advanced MF. The approved JAK inhibitor is not selective for JAK2 but inhibits both JAK1 and JAK2. While effective in reducing patients symptoms associated with MF, JAK1/JAK2 inhibitors frequently cause suppression of platelets and red blood cells, often leading to a need for red blood cell transfusions. Pacritinib may offer an advantage over other JAK inhibitors by having less bone marrow suppression. Such agents may also lead to a modification of the underlying disease process by selectively affecting the malignant clone expressing JAK2V617F.

About Myeloproliferative Neoplasms (MPN)

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Cell Therapeutics Enters into Agreement to Acquire Pacritinib, a Novel Highly Selective JAK2 Inhibitor Phase 3 ...

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18-04-2012 07:24 Many of our patients travel to Guangzhou from all over the world for medical treatment and tourism. China medical tourism can help with becoming a patient, travel arrangements and language assistance. If you want to know more about our services, please browse the or mail to us: Name: Daniel Gender: Male Nationality: USA Age: 50 Start of treatment: 2009-08-12 Diagnosis: brain stem injury Treatment: 3 injections of bone marrow stem cells and nutritious stem cell cocktail treatment Days admitted to the hospital: 40 Reason for coming to treatment: In 1986, Daniel was involved in a car accident, which left him comatose and with little chance of survival. Against the odds Daniel came out of the coma and survived. Today Daniel has limited, but manageable, ability to communicate and to move his body. Wishing to gain more improvements in his motor abilities and improve his quality of life, Daniel and his wife, LaNea, came to Guangzhou for stem cell treatment on September 2009.

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China medical tourism Brain Injury stem cell therapy 2 clip - Video

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18-04-2012 08:35 Many of our patients travel to Guangzhou from all over the world for medical treatment and tourism. China medical tourism can help with becoming a patient, travel arrangements and language assistance. If you want to know more about our services, please browse the web: or mail to us: Name: Shana Weil Gender: Feale Nationality: USA Age: 22 Start of treatment: 2008-11-24 Diagnosis: Traumatic brain injury Treatment: 4 stem cells injections via lumbar puncture and a nutritious stem cell cocktail treatment Days admitted to the hospital: 60 Reason for coming to treatment: On November 20, 2003, Shana lost control of her car and ran into a tree. The accident left her severely brain damaged. Later, Shana came out of the coma, and progressed from a vegetative state to being minimally conscious. Her parents decided to keep their daughter at home, taking care of all her daily needs and kept her on an intense physiotherapy program, but eventually realized that they had exhausted all treatment options available in the US Upon Hearing on the TV about another family that traveled to China for stem cell treatment, her parents decided to travel with their daughter to Guangzhou.

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18-04-2012 09:47 Many of our patients travel to Guangzhou from all over the world for medical treatment and tourism. China medical tourism can help with becoming a patient, travel arrangements and language assistance. If you want to know more about our services, please browse the web: or mail to us: Name: Sammy Ashraf Gender: Female Nationality: Pakistan Age: 32 Start of treatment: 2010-11-18 Diagnosis: Hypoxic encephalopathy, secondary epilepsy Treatment: 3 injections of bone marrow stem cells and nutritious stem cell cocktail treatment Days admitted to the hospital: 30 Reason for coming to treatment: Sammy developed breathing problems and fell into a coma after taking some cough medicine on March 25, 2009, and then she received a tracheotomy, mechanical ventilation to assist her breathing and organic phosphorus detoxification treatment. On the third day, she was taken off the ventilation and continued her treatment in the ICU. She received physical rehabilitation bedside with the assistance of her family members. Three months later, Sammy could understand simple instructions given to her. She was able to swallow small amounts of liquid and move her arms and legs slightly. At the onset of the disease, Sammy was presented with paroxysmal convulsions, clenched fists, and she would grit her teeth. Sammy was put on Topamax for treatment. They looked for new therapy, so they contact us and came to our hospital for ...

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China medical tourism Brain Injury stem cell therapy 4 clip - Video

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18-04-2012 10:06 Many of our patients travel to Guangzhou from all over the world for medical treatment and tourism. China medical tourism can help with becoming a patient, travel arrangements and language assistance. If you want to know more about our services, please browse the web: or mail to us: Name: Binca Karprioru Gender: Female Nationality: Romania Age: 18 Start of treatment: 2007-11-17 Diagnosis: Cerebral Palsy, Mental Retardation Treatment: Stem cell treatment Days admitted to the hospital: 60 Reason for coming to treatment: Due to lack of oxygen during child birth Bianca was born with cerebral palsy characterized mainly by mental retardation. Before the treatment Bianca was very dependent on her mother in all daily activities - eating, dressing, tying her shoes, going to the bathroom and walking. Bianca could walk only with someone supporting her on one side and holding her hand. She could point to something but could not grasp small objects. She could pronounce a few words but was unable to create sentences. She could understand commands of medium difficulty. She appeared to have double vision and had difficulties focusing her sight. She was very anxious around people she didn't know and in unfamiliar environments and would express fear and uncontrolled aggression in such situations. She did not want to miss any opportunity, so she came here in Guangzhou for treatment.

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China medical tourism Cerebral Palsy stem cells therapy 6 clip - Video

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18-04-2012 10:54 Many of our patients travel to Guangzhou from all over the world for medical treatment and tourism. China medical tourism can help with becoming a patient, travel arrangements and language assistance. If you want to know more about our services, please browse the web: or mail to us: Name: Antilla Lawrence Gender: Male Nationality: Hungary Age: 27 Start of treatment: 2008-08-23 Diagnosis: Stem cell treatment Treatment: Weekly stem cell injections, physical therapy, cocktail medication Days admitted to the hospital: 40 Reason for coming to treatment: Antilla suffers from limited mobility and impaired balance due to limited oxygen supply to his brain during his birth. Before coming to Guangzhou Antilla suffered constantly from stiff and rigid muscles. His balance was poor; he could not get down the stairs without catching the handrail, he could not walk very fast and his walking was bumpy and uncoordinated. He could not bend his right knee while walking and used to lean forward with his upper body, a posture which worsened his sense of balance. He used to tire quickly from walking and suffered from back aches after a relatively long walk. He suffered from painful cramps in his legs, mostly during the night. In addition, Antilla's right arm and hand had very limited movement, and since he is right handed, this had a vast effect on his daily activities. He could not straighten his right arm more than ...

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China medical tourism Cerebral Palsy stem cells therapy 7 clip - Video

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18-04-2012 12:47 Many of our patients travel to Guangzhou from all over the world for medical treatment and tourism. China medical tourism can help with becoming a patient, travel arrangements and language assistance. If you want to know more about our services, please browse the web: or mail to us: Name: Garbo Bocskai Gender: Male Nationality: Hungary Age: 20 Start of treatment: 2007-08-30 Diagnosis: Cerebral palsy Treatment: Stem cell treatment Days admitted to the hospital: 50 Reason for coming to treatment: Garbo was born as a result of a premature birth after only 7.5 months of pregnancy, and he suffered Septicemia secondary to Gastroenteritis caused by Thrush only 13 days after birth. Once the infection had been resolved, the infant appeared to have inconsistent bilateral eye movements and paralysis of the lower limbs and was eventually diagnosed with a Brain Injury at the age of 6 months. His parents heard that here in Guangzhou, Gabor would receive the advanced stem cell treatment done by a very professional medical team.

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Public release date: 18-Apr-2012 [ | E-mail | Share ]

Contact: David Eve celltransplantation@gmail.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Tampa, Fla. (April. 18, 2012) Autologous (self-donated) mononuclear cells derived from bone marrow (BMMNCs) have been found to significantly induce vascular growth when transplanted into patients with diabetes who are suffering from critical limb ischemia caused by peripheral artery disease (PAD), a complication of diabetes. The team of researchers in Seville, Spain who carried out the study published their results in a recent issue of Cell Transplantation (20:10), now freely available on-line at http://www.ingentaconnect.com/content/cog/ct/.

"Critical limb ischemia in diabetic patients is associated with high rates of morbidity and mortality; however, neovascularization induced by stem cell therapy could be a useful approach for these patients," said study corresponding author Dr. Bernat Soria of the Andaluz Center for Biologic and Molecular Regenerative Medicine in Seville, Spain. "In this study we evaluated the safety and efficacy of inter-arterial administration of autologous bone marrow-derived mononuclear cells with 20 diabetic patents with severe below-the-knee arterial ischemia."

The researchers noted that surgical or endovascular revascularization options for patients such as those in the study are limited because of poor arterial outflow. Although optimum dose, source and route of administration were outstanding questions, proper BMMNC dose for best results was an issue that the researchers hoped to clarify. They subsequently used a dose ten times smaller than other researchers had used previously in similar studies.

According to the authors, the rationale for their study was that intra-arterial infusions of autologous BMMNCs contain endothelial progenitors that are locally profuse at severely diseased vascular beds in the lower limb. Their hope was that the BMMNCs could promote early and effective development of new vascularization.

Patients were evaluated at three months and twelve months post-transplantation.

"As previously reported, the one-year mortality rate for diabetic patients with PAD - most of which are associated with cardiac complications - has been found to be 20 percent," explained Dr. Soria. "Our study documented significant increases in neovasculogenesis for the majority of our study patients and a decrease in the number of amputations. However, overall PAD mortality for our patients was similar to that generally experienced."

The researchers concluded that BMMNC therapy for lower limb ischemia was a "safe procedure that generates a significant increase in the vascular network in ischemic areas" and promotes "remarkable clinical improvement."

"While this study did not demonstrate a significant effect on mortality, it does suggest an improvement in the quality of life based on limb retention as shown by the significant reduction in the number of amputations", said Amit N. Patel, director of cardiovascular regenerative medicine at the University of Utah and section editor for Cell Transplantation.

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Autologous bone marrow-derived mononuclear cell transplants can reduce diabetic amputations

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There has been no shortage of baldness cures over the ages, but they all share one thing in common: none of them really work. Now, a team of scientists has used stem cell therapy to give a hairless mouse a mohawk. There is hope yet.

The researchers, from the Tokyo University of Science, have seized on the concept of using stem cells to provide regenerative medicine, and given it a twist. Actually, maybe more of a curl, because they hit on the idea of using the therapyusually reserved for restoring organs damaged by disease or illnessto regenerate hair follicles.

To do that, they created a "seed" of a hair follicle by combining adult epithelial stem cells and dermal papilla cellstwo basic cells that are found in the skinfrom a normal mouse. Then, they inserted that seed into the skin of a hairless mouse, which are genetically engineered for just this kind of research, and... waited.

The result? Fully functional hair follicles, that grew a respectable amount of hair. Not just that, these things connected properly with the skin and nerves, went through the typical cycle of shedding hairs and then regrowing, and could even get goosebumps. These hair follicles are the real deal. The research is published in Nature Communications.

All told, it's perhaps the most promising solution to regenerating hair that we've seen. The catchthere's always a catch, right?is that, as yet, it's entirely untested in humans. And even if it was, this kind of therapy is extremely exotic, so wouldn't come cheap. [Nature Communications]

Image by Tokyo University of Science

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Can Stem Cells Finally Provide a Baldness Cure That Works? [Science]

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From Rogaine to hair plugs, there have been many strides made to induce hair regrowth for bald or balding heads, but a new Japanese study found a possible cure to baldness by using stem cells.

Using stem cell therapy, scientists at the Tokyo University of Science in Japan led by Takashi Tsuki gave a hairless mouse a Mohawk by regenerating hair follicles.

Researchers used follicles from a normal mouse, namely adult epithelial stem cells and dermal papilla cell found in the skin, to create a "seed" of a hair follicle. Then, they implanted the newly-created "seed" using intracutaneous transplantation into the hairless mouse and -- Voila! -- hair.

According to the research published in Nature Communications, functional hair follicles grew on the hairless mouse properly on the skin in the epidermis, arrector pili muscle and nerve fibers. The newly regrown hair also went through a standard hair cycle of shedding and regrowth.

"Our current study thus demonstrates the potential for not only hair regeneration therapy but also the realisation of bioengineered organ replacement using adult somatic stem cells," the report said.

The baldness cure that worked on the hairless mice, however, has not yet been tested on humans, but the researchers hope to introduce the idea soon.

"We would like to start clinical research within three to five years, so that an actual treatment to general patients can start within a decade," researcher Koh-ei Toyoshima said in a statement.

However, even if it does work on people, the issue is raised about the cost, as stem cell therapy practices can be quite costly.

View the video of the hairless mouse with hair regrowth below.

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Baldness Cure: Japanese Study Finds Stem Cells Induce Hair Regrowth for Bald Heads on Mouse [PHOTO & VIDEO]

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Public release date: 18-Apr-2012 [ | E-mail | Share ]

Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center

STANFORD, Calif. Heart-like cells made in the laboratory from the skin of patients with a common cardiac condition contract less strongly than similarly created cells from unaffected family members, according to researchers at the Stanford University School of Medicine. The cells also exhibit abnormal structure and respond only dully to the wave of calcium signals that initiate each heartbeat.

The finding used induced pluripotent stem, or iPS, cell technology to create heart-muscle-like cells from the skin of patients with dilated cardiomyopathy, which is one of the leading causes of heart failure and heart transplantation in the United States. It adds to a growing body of evidence indicating that iPS cells can faithfully reflect the disease status of the patients from whom they are derived.

Using the newly created diseased and normal cells, the researchers were able to directly observe for the first time the effect of a common beta blocker drug, as well as validate the potential usefulness of a gene therapy approach currently in clinical trials.

"Primary human cardiac cells are difficult to obtain and don't live long under laboratory conditions," said Joseph Wu, MD, PhD, associate professor of cardiovascular medicine. Instead, researchers have relied on studies of cells from rodent hearts, which beat much more quickly, to understand more about human heart disease. "Now we've created heart cells from iPS cells derived from skin that allow us to study in detail the mechanisms of a common cardiac disease and how these cells respond to clinical interventions."

Wu is the senior author of the research, which will be published April 18 in Science Translational Medicine. Postdoctoral scholar Ning Sun, MD, PhD, is the first author. The work is the latest in a type of research that's sometimes referred to as "disease-in-a-dish" studies. Using iPS technology, other researchers have created stem cells from patients with Parkinson's disease, Marfan syndrome and amyotrophic lateral sclerosis, among others.

The implications of such research are huge. According to Wu, one of the major reasons cardiac drugs are pulled from the market is unexpected cardiac toxicity that is, they are damaging the very hearts they're meant to help. Currently, such drugs are pre-screened for toxic effects on common laboratory cell lines derived from either hamster ovaries or human embryonic kidney cells. Even though these ovarian and kidney cells have been artificially induced to mimic the electrophysiology of human heart cells, they are still very different from the real thing. A reliable source of diseased and normal human heart cells on which to test the drugs' effect prior to clinical use could improve drug screening, save billions of dollars and improve the lives of countless patients.

Dilated cardiomyopathy occurs when a portion of the heart muscle enlarges and begins to lose the ability to pump blood efficiently. Eventually, the enlarged muscle begins to weaken and fail, requiring either medication or even transplant. Although many cases occur sporadically and without an apparent cause, dilated cardiomyopathy can also be inherited via a variety of genetic mutations.

Wu and Sun performed skin biopsies on seven members of three generations of a family with the inherited form of the condition (called familial dilated cardiomyopathy). Four of the family members had inherited a specific genetic mutation in a gene called TNNT2 that causes the disease; the other three had not. The researchers used iPS technology to convert skin cells from the affected and unaffected family members into stem cells, which they then coaxed to become heart muscle cells for further study. They then compared cells from unaffected family members with those who had the disease.

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Stanford scientists show lab-made heart cells ideal for disease research, drug testing

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THE WOODLANDS, Texas--(BUSINESS WIRE)--

Opexa Therapeutics, Inc. (NASDAQ: OPXA - News), a biotechnology company developing Tovaxin, a novel T-cell therapy for multiple sclerosis (MS), announced today that the Company will be holding a preliminary meeting with prospective clinical trial investigators at the 64th Annual American Academy of Neurology (AAN) Meeting in New Orleans on April 24, 2012. The purpose of this meeting will be to discuss the upcoming Phase IIb clinical trial using Opexas T-cell therapy in patients with Secondary Progressive Multiple Sclerosis (SPMS).

"We are honored to introduce our next clinical trial to a group of invited neurologists at this years AAN meeting in New Orleans, commented Neil K. Warma, President and Chief Executive Officer of Opexa. The meeting will be an opportunity to discuss with select clinicians and their study coordinators potential participation in the SPMS clinical trial as well as present the final protocol for the trial including the design, structure and patient selection criteria. This is an exciting time for Opexa, neurologists and SPMS patients as this study will provide an innovative opportunity for treatment in an area where currently there are very few treatment options. The annual AAN meeting is an excellent forum for the MS community to discuss and present new therapies that could have an important impact on the treatment of MS. We are pleased that Tovaxin is generating a great deal of enthusiasm among physicians, key opinion leaders and patients in the lead up to this meeting and are equally pleased to be advancing our clinical plans for Tovaxin.

Mark Freedman, M.D., director of the Multiple Sclerosis Research Unit at the Ottawa Hospital and member of Opexas Scientific Advisory Board, commented, I am pleased to contribute my expertise to Opexa with their design and planning of this Phase IIb study. Opexa is now in the process of selecting clinical trial investigators and finalizing the remaining steps in order to conduct a study of optimal quality. I am pleased to facilitate the introduction of the Phase IIb study to prospective clinical trial investigators at this years AAN meeting. Patients with SPMS have few treatment options and Tovaxins safety profile certainly justifies investigation of this therapy in the challenging SPMS patient population.

The proposed Phase IIb clinical trial will be a randomized, double-blind, placebo-controlled study of Opexas T-cell therapy in SPMS patients with evidence of disease progression without associated relapses. The study, to be initiated once the necessary resources are secured, is expected to treat approximately 180 patients in up to 30 sites in the United States and Canada with annual courses of treatment for two years.

About Opexa

Opexa Therapeutics, Inc. is dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS). The Companys leading T-cell therapy, a personalized cellular immunotherapy treatment, is in clinical development targeting both Secondary Progressive and Relapsing Remitting MS. Opexas T-cell therapy is derived from T-cells isolated from peripheral blood, expanded ex vivo and reintroduced into the patients via subcutaneous injections. This process triggers a potent immune response against specific subsets of autoreactive T-cells known to attack myelin and, thereby, reduces the risk of relapse over time.

For more information, visit the Companys website at http://www.opexatherapeutics.com.

Cautionary Statement Relating to Forward - Looking Information for the Purpose of "Safe Harbor" Provisions of the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The words expects, believes, anticipates, estimates, may, could, intends, and similar expressions are intended to identify forward-looking statements. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding the development of the Companys product candidate, Tovaxin, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: our capital position, the ability of the Company to enter into and benefit from a partnering arrangement for the Company's product candidate, Tovaxin, on reasonably satisfactory terms (if at all), our dependence (if partnered) on the resources and abilities of any partner for the further development of Tovaxin, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of developing a marketable product, our ability to raise additional capital to continue our treatment development programs and to undertake and complete any further clinical studies for Tovaxin, the success of our clinical trials, the efficacy of Tovaxin for any particular indication, such as Relapsing Remitting MS or Secondary Progressive MS, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights (including for Tovaxin), the risk of litigation regarding our intellectual property rights, the success of third party development and commercialization efforts with respect to products covered by intellectual property rights that the Company may license or transfer, our limited manufacturing capabilities, our dependence on third-party manufacturers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in our filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date made. We assume no obligation or undertaking to update any forward-looking statements to reflect any changes in expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based. You should, however, review additional disclosures we make in our reports filed with the Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2011.

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Opexa to Hold Preliminary Investigator’s Meeting for MS Trial at American Academy of Neurology Annual Meeting

Recommendation and review posted by Bethany Smith

Researchers from the Gladstone Institutes in the US showed for the first time that injecting a combination of genes into the damaged heart tissue of a living animal could make it beat again.

Although the technique is a long way from being fit for human trials, experts said it could eventually lead to a way of repairing the debilitating damage sustained in heart attacks.

The results, published in the Nature journal, replicate previous test tube studies which suggested that the three genes, which normally guide the development of the heart in embryos, could reprogram non-beating cells into fully functional heart muscle.

Dr Deepak Srivastava, who led the study, said: "These findings could have a significant impact on heart-failure patients, whose damaged hearts make it difficult for them to engage in normal activities like walking up a flight of stairs."

Prof Peter Weissberg, Medical Director of the British Heart Foundation, added: This research illustrates one of many routes scientists are exploring to try and repair damage caused by a heart attack.

"If this is confirmed by further studies, it is a remarkable achievement. But a great deal more research will be needed before we will know whether such an approach is feasible, or indeed safe, in patients.

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Heart damage repaired in mice by gene therapy

Recommendation and review posted by Bethany Smith

Public release date: 18-Apr-2012 [ | E-mail | Share ]

Contact: Vanessa Pavinato media@esmo.org European Society for Medical Oncology

Lugano-CH/Aurora-US-CO/Geneva-CH, 18 April 2012 -- A new genetic signature identified by Spanish researchers may provide doctors with robust and objective information about which patients with early stage lung cancer are at low or high risk of relapse following surgery, investigators report at the 3rd European Lung Cancer Conference in Geneva. Their work also opens new avenues for immunotherapy for lung cancer.

Non-small cell lung cancer is a disease that is often not diagnosed until it has grown and spread throughout the body. Even those patients who are diagnosed early enough to undergo surgical removal of the tumor still have a discouraging 30% rate of relapse.

Researchers hope that identifying which patients have the greatest risk of relapse will allow doctors to focus other treatment strategies, in order to improve their chance of being cured after surgery.

A multidisciplinary team of researchers from Hospital Clinico San Carlos, Madrid, have found a 50-gene predictor that appears to be capable of doing just that. In a study of 84 patients with stage I and II non-small cell lung cancer, who had undergone surgery to remove their tumor, the gene signature accurately predicted which patients were at low risk of relapse.

The researchers analyzed genetic expression in the tumor mass using microarray technology. Following patients for six years, they were able to correlate gene expression patterns with the clinical course of disease, and the risk of relapse.

The Spanish research team's report at the meeting shows that the genes of the predictor were over-expressed in roughly one-third of patients, all of whom had a low risk of relapse. Further analysis showed that these genes were related to the activity of important immune system white blood cells, known as B lymphocytes.

"All of these genes overexpressed in the low-risk group are highly related to B lymphocyte activity," said Dr Florentino Hernando presenting the results at the meeting. "So, the B cell-mediated immune response seems to have a very important role."

The genetic profile identified by the researchers suggests that low-risk patients have an enhanced immune response against the tumor. "Thus, treatments that may interfere with this response such as post-surgical chemotherapy must be reconsidered for the low-risk subgroup," he says.

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Gene signature helps identify risk of relapse in lung cancer patients

Recommendation and review posted by Bethany Smith

A brain study has found a gene linked to intelligence, a small piece in the puzzle as to why some people are smarter than others. shutterstock.com

A variant of this gene can tilt the scales in favour of a higher intelligence, study leader Paul Thompson said, stressing though that genetic blessings were not the only factor in brainpower.

Searching for a genetic explanation for brain disease, the scientists stumbled upon a minute variant in a gene called HMGA2 among people who had larger brains and scored higher on standardised IQ tests.

Thompson dubbed it an intelligence gene and said it was likely that many more such genes were yet to be discovered.

The variant occurs on HMGA2 where there is just a single change in the permutation of the four letters of the genetic code.

DNA, the blueprint for life, comprises four basic chemicals called A (for adenine), C (cytosine), T (thymine) and G (guanine), strung together in different combinations along a double helix.

In this case, the researchers found that people with a double C and no T in a specific section of the HMGA2 gene had bigger brains on average.

It is a strange result, you wouldnt think that something as simple as one small change in the genetic code could explain differences in intelligence worldwide, said Thompson, a neurologist at the University of California at Los Angeles.

The discovery came in a study of brain scans and DNA samples from more than 20,000 people from North America, Europe and Australia, of European ancestry.

People who received two Cs from their parents, a quarter of the population, scored on average 1.3 points higher than the next group half of the population with only one C in this section of the gene.

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Tiny gene change affects brain size, IQ: Scientists

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ScienceDaily (Apr. 19, 2012) Scientists led by Washington University School of Medicine in St. Louis have identified the first gene directly linked to the most common form of psoriasis, a chronic skin condition.

The research shows that rare mutations in the CARD14 gene, when activated by an environmental trigger, can lead to plaque psoriasis. This type of psoriasis accounts for 80 percent of all cases and is characterized by dry, raised, red patches covered with silvery scales that can be itchy and painful.

The new findings also indicate that mutations in CARD14 can be involved in the pustular form of psoriasis and in a debilitating arthritis linked to the psoriasis. The discovery may lead to more effective, targeted therapies for plaque psoriasis and other forms of the disease.

The research is published May 4 in two separate papers in The American Journal of Human Genetics.

"We have searched for almost two decades to find a single gene linked to plaque psoriasis," says the senior author of both papers, Anne Bowcock, PhD, professor of genetics. "Individually, the rare mutations we have found likely confer a high risk for the disease, and we think they will be important in the search to find new, more effective treatments."

Although psoriasis has long been thought to be caused by an overactive immune system, the genetic pathway uncovered by the scientists points to defects in the skin as the main culprit of the condition and to immune cells as secondary players.

Now, the researchers want to find out how common the altered pathway is in the different types of psoriasis and in patients with psoriatic arthritis. Their work suggests that in at least some patients with different forms of psoriasis, this pathway is the same.

An estimated 7.5 million Americans have psoriasis, and about 30 percent of them develop psoriatic arthritis. Like other common diseases, psoriasis runs in families and has been thought to have a genetic component, but it's been difficult to pin down the genes involved. That's because common variations in genes likely contribute very little to the overall genetic risk of the disease, and mutations that substantially increase a person's risk are so rare they have been impossible to find.

With early support from the National Psoriasis Foundation, Bowcock initiated the research with co-author Alan Menter, MD, of the Psoriasis Research Institute at the Baylor College of Medicine.

Using the latest DNA technology to sequence all of a patient's genes, Bowcock and her colleagues uncovered a rare CARD14 mutation in a large family of northern European descent in which plaque psoriasis was prevalent. They also found the mutation in the one-third of family members who had developed psoriatic arthritis, suggesting that the same rare mutation can play a role in both conditions.

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First gene linked to common form of psoriasis identified

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PISCATAWAY, N.J., April 19, 2012 /PRNewswire-Asia/ -- GenScript USA Inc., an internationally recognized biology contract research organization (CRO) has recently launched Rush Gene Synthesis service in the global market on Dec. 22, 2011, and has achieved 100% success and on time delivery rates ever since its kick-off.

"Two decades ago, a 2 Kb gene took me nearly two years to obtain the construct using conventional PCR cloning technologies in my PhD research. I did believe this could be changed. Nowadays, GenScript Rush Gene Synthesis service delivers synthetic genes in as little as 5 business days. The proprietary technology and a dedicated Rush Gene service team are the key points to making GenScript Rush Gene Synthesis service a big success," commented Frank Zhang, the CEO and co-founder of GenScript.

"Delivering synthetic genes in as little as 5 days, GenScript Rush Gene Synthesis service is not only fast but is accurate and worry-free to our customers," says Frank, "all our synthesized genes are packed into cloning vectors, picked from single clones and fully sequence-verified before the delivery. Therefore, none of our customers is bothered with picking up the right gene from the tube. With this rush service, we aim to drive molecular biology research faster than ever."

Besides the world-leading gene synthesis service, GenScript, as a contract research organization (CRO), also provides comprehensive services for biological research and early-phase drug discovery, such as bio-reagents, assay development & screening, lead optimization, antibody drug development and animal model services. The bio-reagents services include custom gene synthesis and molecular biology, custom protein expression and purification, custom peptide synthesis, antibody production, and custom cell line development. Headquartered in Piscataway, New Jersey, GenScript has three subsidiaries located in France, Japan, and China.

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GenScript Rush Gene Synthesis - Driving Molecular Biology Research Faster

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Public release date: 18-Apr-2012 [ | E-mail | Share ]

Contact: Michael Bernstein m_bernstein@acs.org 202-872-6042 American Chemical Society

Scientific insights that expand on the teachings of Mendel, Watson and Crick, and underpinnings of the Human Genome Project are moving drug companies along the path to development of new medicines based on deeper insights into how factors other than the genetic code influence health and disease. That's the topic of the cover story in the current edition of Chemical & Engineering News (C&EN), the weekly newsmagazine of the American Chemical Society (ACS), the world's largest scientific society.

The article, by C&EN Senior Editor Lisa M. Jarvis, focuses on the quiet revolution in epigenetics that has been sweeping through biology, chemistry and other scientific fields for the last several years. It explains how scientists initially believed that cracking the genetic code, achieved a decade ago, would lay out a straight path for inventing new medicines: Identify the genetic mutation behind a disease and then find a drug that overcomes it. But scientists now know that another layer of biochemical controls, an epigenetics layer, influences how and when genes work in health and disease without changing DNA itself. Early epigenetics research already produced four drugs currently approved to treat blood cancer. But these treatments lack selectivity, limiting their effectiveness.

Now, Jarvis explains, companies like GlaxoSmithKline, Epizyme and Constellation Pharmaceuticals are moving ahead to develop the next generation of epigenetic drugs, particularly for cancer. Armed with a better understanding of how specific epigenetic enzymes are implicated in disease, they are designing compounds to block the activity of those enzymes. The article describes GSK's announcement earlier this month of an epigenetic inhibitor it has developed that might fight lymphoma. "Although no one will know the value of the new epigenetic compounds until they are tested in humans, scientists are confident that the field is moving forward with the right balance of caution and enthusiasm," Jarvis concludes.

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The American Chemical Society is a nonprofit organization chartered by the U.S. Congress. With more than 164,000 members, ACS is the world's largest scientific society and a global leader in providing access to chemistry-related research through its multiple databases, peer-reviewed journals and scientific conferences. Its main offices are in Washington, D.C., and Columbus, Ohio.

To automatically receive news releases from the American Chemical Society contact newsroom@acs.org.

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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Not by DNA alone: How the epigenetics revolution is fostering new medicines

Recommendation and review posted by Bethany Smith

ST. LOUIS, April 19, 2012 /PRNewswire/ --Sigma-Aldrich Corporation (Nasdaq: SIAL - News) today announced that Sigma Advanced Genetic Engineering (SAGE) Labs, an initiative of Sigma Life Science, extended CompoZr Zinc Finger Nuclease (ZFN) technology to achieve the first tissue-specific conditional knockout of an endogenous gene in rats. For two decades this approach for generating sophisticated disease models could be performed only in mice. Rats, however, are preferred by drug discovery and basic researchers because the animal's physiology, neurobiology and other features are more predictive of human conditions. Rats engineered to contain tissue-specific conditional gene knockouts are available exclusively through the SAGEspeed Custom Model Development Service. Details are available at http://www.sageresearchmodels.com/conditional-KO.

(Logo: http://photos.prnewswire.com/prnh/20050215/CGSIGMAALLOGO)

Conventional gene knockout eliminates a gene throughout an entire animal. In contrast, conditional gene knockout can eliminate a gene solely in the relevant tissue or organ, leading to a more accurate understanding of the gene's function. Conditional gene knockout can also knock out genes at certain points in development, enabling studies of genes whose absence in embryos is lethal, but whose loss of function in adulthood is critical to investigate for many human diseases.

"Almost 89% of drug candidates fail to achieve approval," said Edward Weinstein, Director of SAGE Labs. "Basic and drug discovery researchers need access to more predictive animal models whose physiology, biology, and genetics more closely reflect specific human conditions. SAGE Labs is applying ZFN technology to achieve previously impossible genetic manipulations, such as tissue-specific gene deletion in rats."

Using the conditional knockout methodology, scientists at SAGE Labs have generated a pair of rat lines in which two important neuronal genes, Crhr1 and Grin1, were removed in specific neuronal populations. Crhr1 and Grin1 have been implicated as playing a role in depression and schizophrenia, respectively. The rat lines were developed through the SAGEspeed model creation process, which uses Sigma's CompoZr ZFN technology to create sophisticated genetic modifications in rats, mice, rabbits, and other organisms. CompoZr ZFN technology is the first to enable highly efficient, targeted editing of the genome of any species.

For more information and to request pricing, visit http://www.sageresearchmodels.com.

Cautionary Statement: The foregoing release contains forward-looking statements that can be identified by terminology such as "enable," "enabling," "leading to," "achieve," "predictive" or similar expressions, or by expressed or implied discussions regarding potential future revenues from products derived there from. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that iPS cells, iPS-cell derived primary cell lines, novel assays, or related custom services will assist the Company to achieve any particular levels of revenue in the future. In particular, management's expectations regarding products associated iPS cells, iPS-cell derived primary cell lines, novel assays, or related custom services could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; the Company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Company's assets and liabilities as recorded in its consolidated balance sheet, and other risks and factors referred to in Sigma-Aldrich's current Form 10-K on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Sigma-Aldrich is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Sigma Life Science: Sigma Life Science is a Sigma-Aldrich business that represents the Company's leadership in innovative biological products and services for the global life science market and offers an array of biologically-rich products and reagents that researchers use in scientific investigation. Product areas include biomolecules, genomics and functional genomics, cells and cell-based assays, transgenics, protein assays, stem cell research, epigenetics and custom services/oligonucleotides. Sigma Life Science also provides an extensive range critical bioessentials like biochemicals, antibiotics, buffers, carbohydrates, enzymes, forensic tools, hematology and histology, nucleotides, amino acids and their derivatives, and cell culture media.

About Sigma-Aldrich: Sigma-Aldrich is a leading Life Science and High Technology company whose biochemical, organic chemical products, kits and services are used in scientific research, including genomic and proteomic research, biotechnology, pharmaceutical development, the diagnosis of disease and as key components in pharmaceutical, diagnostics and high technology manufacturing. Sigma-Aldrich customers include more than 1.3 million scientists and technologists in life science companies, university and government institutions, hospitals and industry. The Company operates in 40 countries and has nearly 9,000 employees whose objective is to provide excellent service worldwide. Sigma-Aldrich is committed to accelerating customer success through innovation and leadership in Life Science and High Technology. For more information about Sigma-Aldrich, please visit its website at http://www.sigma-aldrich.com.

Sigma-Aldrich and Sigma are trademarks of Sigma-Aldrich Co, LLC registered in the US and other countries. SAGE and CompoZr are registered trademarks of Sigma-Aldrich Co. LLC. SAGEspeed is a trademark of Sigma-Aldrich Co. LLC.

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SAGE® Labs Creates the First Tissue-Specific Gene Deletion in Rats

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A team of UCLA researchers has found a genetic engineering technique that suppresses the HIV virus in mice, an encouraging step toward potentially fighting the disease in humans, researchers said.

The study, led by Scott Kitchen, a member of the UCLA AIDS Institute and assistant professor at the David Geffen School of Medicine, draws upon previous UCLA research findings. It was published last week in the epidemiology journal PLoS Pathogens and funded in part by the UCLA Center for AIDS Research.

Researchers examined the effectiveness of genetically engineered killer T cells, which are capable of fighting off disease, at combating the HIV virus in a mouse. The team used a humanized mouse engineered to have a human immune system. In the humanized mouse, the disease progressed similarly to its progression in humans, making it a reliable tool for the study and providing powerful predictive value for the therapy in humans, Kitchen said. Its a major advance and a step closer in demonstrating the potential use of this in people, he said.

A few months ago, the team introduced a population of the engineered T cells into a mouse so they could develop and grow into a human immune system, Kitchen said. The researchers then conducted blood and organ tests at the second and sixth weeks, finding a decrease in the HIV levels and an increase in the cells HIV typically kills, according to the journal article.

The findings could theoretically be used to support a clinical trial in humans, said Jerome Zack, associate director at the UCLA AIDS Institute and co-author of the study.

A benefit genetic engineering is that it opens the field to therapeutic HIV treatments, and that it can be extended to potentially treat other diseases such as cancer, said co-author Arumugam Balamurugan.

In 2009, the lead scientists from the most recent study showed that human blood stem cells in mice could be genetically engineered to grow large quantities of killer T cells, As a result of their genetic engineering, these T cells grew to a large population and targeted HIV-infected cells in the mice.

We had the idea that we could take the elements of immune response (the T cells) that are successful in suppressing HIV in infected people to see if it was possible to identify a receptor specific to HIV, Kitchen said, referring to the teams research in 2009.

Though advances have been made in the fight against HIV, an estimated 50,000 new cases are diagnosed in the United States each year, and there are more than 33 million people living with the disease worldwide, according to the National Institutes of Health. Factors that make the disease difficult to fight include its rapid rate of spread and lack of preventative measures. The findings could lead to more comprehensive methods of fighting the disease and eventually to a clinical trial in humans, Kitchen said.

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UCLA researchers develop genetically engineered stem cells to fight HIV in mice

Recommendation and review posted by Bethany Smith