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Sixteenth Patient Dosed In Neuralstem ALS Stem Cell Trial

ROCKVILLE, Md., June 19, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that the first patient to receive stem cell transplantation in both regions of the spinal cord has been treated in the ongoing Phase I trial of its spinal cord neural stem cells in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). This is also the 16th patient to be treated in the trial altogether and the first patient returning to the trial for a second treatment. In this treatment, the patient received five injections in the cervical (upper back) region of the spinal cord, in addition to the ten he received previously in the lumbar (lower back) region of the spine, for a total of 15 injections. This is the highest number of injections in the trial so far. Patient 16 is also the first patient in the world to receive stem cell transplants in both the lumbar and cervical regions of the spinal cord in an FDA-approved trial. Two additional previously-treated patients are expected to return to the trial this summer in this cohort, provided they continue to meet the inclusion requirements. The trial is taking place at Emory University Hospital in Atlanta, Georgia.

(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )

"Transplanting the first of the returning patients represents a major milestone in the trial," said Dr. Karl Johe, PhD, Neuralstem's Chairman and Chief Scientific Officer. "The ability to safely administer multiple dosings to these patients is a key enabling step in administering the maximum safe dose. Not only are we dosing patients for a second time in this cohort, we are now dosing in both the lumbar and cervical regions of the spinal cord for the first time, where the stem cell therapy could support both walking and breathing."

About the Trial

The Phase I trial to assess the safety of Neuralstem's spinal cord neural stem cells and intraspinal transplantation method in ALS patients has been underway since January 2010. The trial is designed to enroll up to 18 patients. The first 12 patients were each transplanted in the lumbar (lower back) region of the spine, beginning with non-ambulatory and advancing to ambulatory cohorts.

The trial then advanced to transplantation in the cervical (upper back) region of the spine. The first cohort of three was treated in the cervical region only. The current cohort of three will receive injections in both the cervical and lumbar regions of the spinal cord. In an amendment to the trial design, The Food and Drug Administration (FDA) approved the return of previously-treated patients to this cohort. The first of these returning patients was just treated. The entire 18-patient trial concludes six months after the final surgery.

About Neuralstem

Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem is in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.

In addition to ALS, the company is also targeting major central nervous system conditions with its cell therapy platform, including spinal cord injury, ischemic spastic paraplegia and chronic stroke. The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in chronic spinal cord injury.

Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain's capacity to generate new neurons, possibly reversing the pathologies of some central nervous system conditions. The company has received approval from the FDA to conduct a Phase Ib safety trial evaluating NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD). Additional indications could include CTE (chronic traumatic encephalopathy), Alzheimer's disease, anxiety, and memory disorders.

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Sixteenth Patient Dosed In Neuralstem ALS Stem Cell Trial

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Bethel woman waits for marrow match

BETHEL, Vt. -

Amelia Lincoln loves to garden. But so far this planting season, she has had to sit it out.

"It's been a long haul and we try to keep a pretty positive attitude about everything. So, I generally could feel worse right now," she said.

Lincoln's immune system is fragile. For the past six weeks, she has been undergoing chemotherapy for cancer.

"I have acute myelogenous leukemia," she said. "I have a leukemia that came back after a stem cell transplant two years ago."

She had been in remission, but the aggressive cancer in her bone marrow is back.

"It's a change of priorities, but what would anyone say if their spouse was sick," husband James Patterson said.

Lincoln needs another transplant at the Norris Cotton Cancer Center. But right now, she has yet to find the perfect match.

"We used to use bone marrow specifically for a bone marrow transplant. Nowadays, we can use medicines to stimulate a patient's bone marrow cells into the blood. We can collect those bone marrow cells in the blood-- called peripheral blood stem cells-- and use those cells for the transplant," said Dr. Kenneth Meehan of the Norris Cotton Cancer Center.

A donor drive Tuesday in Randolph Center could increase Amelia's odds. No needles-- just a swab.

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Bethel woman waits for marrow match

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Stem Cell Therapy for CMT-Gary B-part 2.mp4 – Video

17-06-2012 23:59 Gary B Stem Cell Therapy for CMT Part 2 - For more info. visit

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Full Spectrum Genetics Enters Strategic Collaboration With a Leading Global Pharmaceutical Company

SOUTH SAN FRANCISCO, CA--(Marketwire -06/19/12)- Full Spectrum Genetics, Inc., a privately-held protein analysis and engineering platform and product company, today announced a strategic collaboration with an undisclosed leading global pharmaceutical company for the purpose of generating multiple novel therapeutic protein and antibody product candidates.

Under the terms of the agreement, Full Spectrum Genetics will be responsible for engineering and analyzing variants of specified molecules using its MapEng platform. The pharmaceutical company will receive worldwide rights to develop and commercialize product candidates arising from the collaboration. Full Spectrum Genetics will receive an upfront payment to initiate the collaboration and is eligible for additional financial consideration. Additional terms were not disclosed.

About Full Spectrum Genetics

Founded in 2010, Full Spectrum Genetics, Inc. is a privately-held protein analysis and engineering platform and product company. The Company's MapEng platform enables the ultra-high throughput quantification of the effect on binding of every possible single amino acid substitution within a protein binding site. The MapEng platform provides a comprehensive analysis of protein structure-function relationships, with multiple applications for generating better biotherapeutics and diagnostics. For more information on Full Spectrum Genetics and its MapEng platform, visit http://www.fsgene.com.

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Bethel woman waits for marrow match

BETHEL, Vt. -

Amelia Lincoln loves to garden. But so far this planting season, she has had to sit it out.

"It's been a long haul and we try to keep a pretty positive attitude about everything. So, I generally could feel worse right now," she said.

Lincoln's immune system is fragile. For the past six weeks, she has been undergoing chemotherapy for cancer.

"I have acute myelogenous leukemia," she said. "I have a leukemia that came back after a stem cell transplant two years ago."

She had been in remission, but the aggressive cancer in her bone marrow is back.

"It's a change of priorities, but what would anyone say if their spouse was sick," husband James Patterson said.

Lincoln needs another transplant at the Norris Cotton Cancer Center. But right now, she has yet to find the perfect match.

"We used to use bone marrow specifically for a bone marrow transplant. Nowadays, we can use medicines to stimulate a patient's bone marrow cells into the blood. We can collect those bone marrow cells in the blood-- called peripheral blood stem cells-- and use those cells for the transplant," said Dr. Kenneth Meehan of the Norris Cotton Cancer Center.

A donor drive Tuesday in Randolph Center could increase Amelia's odds. No needles-- just a swab.

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Bethel woman waits for marrow match

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The WIN 4th SYMPOSIUM, on June 28-29 in Paris, Scientific Program Recognized by Leading Oncology Society

PARIS--(BUSINESS WIRE)--

The scientific program of the 4th WIN Symposium, on Cancer personalized medicine and efficacy of biomarkers, a major event where hundreds of representatives from 20 countries will gather on next June 28 and 29, 2012 in Paris (France), has been endorsed by the American Society of Clinical Oncology (ASCO), the worlds leading professional organization representing physicians who care for people with cancer.

John Mendelsohn, Chairman of WIN Consortium and Director of the Institute for personalized therapy, UT M. D. Anderson Cancer Center, says: we have been proud to receive the endorsement of ASCO for the quality of educational content of the WIN Symposium Program 2012. This is a great achievement and recognition of the scientific value of the WIN Program Symposium and the major impact this symposium may bring.

The WIN Consortium meeting reflects the vision and goals of ASCOs blueprint for transforming cancer research (www.ASCO.org/Blueprint) and the potential that molecularly-driven therapies have to transform clinical research, said Sandra Swain, MD, President of ASCO. ASCO is pleased to endorse this meeting because of the opportunities it provides for international collaboration.

Organized by the WIN Consortium, this congress gathers the main opinion leaders in cancer care and drug development for two days of debates and exchanges and offers an excellent opportunity to advance research in Cancer and biomarkers at international level, facilitating the translation of scientific results into clinical practice. Advanced and innovative concepts to increase efficacy of personalized cancer therapeutics and molecular diagnostics will be disclosed during WIN Symposium: launch of WINTHER, the most advanced study in personalized medicine selected by the European Community EU FP7.

About WIN Consortium: The WIN Consortium is an initiative of the Cancer Institute Gustave Roussy, Villejuif, IGR (France) and University of Texas MD Anderson Cancer Center (USA), which brings together 22 international cancer centers, three technology partners (Agilent Technologies, GE Healthcare and LifeTechnologies) and associations such as Sage Bionetworks and the National Breast Cancer Foundation as part of a legal entity operating non-profit. Countries participating in the symposium: U.S., France, Hungary, Romania, Singapore, Brazil, Spain, Italy, India, China, Israel, Jordan, UK, Canada... The american Foundation Medicine is an official member of the WIN Consortium. The quality of the WIN 2012 Symposium program has been also recognized by several major international organizations: ESMO, INCa and UICC.

ASCO is a registered trademark of the American Society of Clinical Oncology. Used with permission. This is not an ASCO sponsored event.

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The WIN 4th SYMPOSIUM, on June 28-29 in Paris, Scientific Program Recognized by Leading Oncology Society

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CepMed Appoints New CEO

MONTREAL, QUEBEC--(Marketwire -06/18/12)- Mr. Michel Saucier, Chairman of the Board of Directors of CepMed, the Center of Excellence in Personalized Medicine, is pleased to announce the appointment of Ms. Manon Decelles, CPA, CGA as Chief Executive Officer. Coincident with Ms. Decelles' arrival, Dr. Clarissa Desjardins, outgoing CEO, is leaving to manage a start-up biotechnology company.

Ms. Decelles has 20 years of experience in the pharmaceutical industry and a proven track record in key areas of business including finance, marketing and business development. Together with this experience Ms. Decelles brings an extensive business network that will enable CepMed in bringing together medical, scientific and business communities to promote innovation and commercialization in personalized medicine. Prior to joining CepMed, Ms. Decelles was Director of Business Development and Acquisitions at Sanofi, a global and diversified healthcare leader, where she was responsible for the Canadian affiliate's business development activities, including in and out licensing, divestitures as well as company acquisitions. In this capacity, Ms. Decelles developed and implemented a Strategic Business Development Plan and review process that guided Sanofi's Canadian strategy. Previously, Ms. Decelles held roles of increasing responsibility in finance, marketing and business development at Marion Merrell Dow, Hoechst Marion Roussel and Aventis. Ms. Decelles is an active member of the Board of Directors of the Canadian Healthcare Licensing Association.

Ms. Decelles first studied in science - microbiology at the University of Sherbrooke. She later obtained a Bachelor of Business Administration from the University of Montreal, HEC and is also a Chartered Professional Accountant (CPA).

"As Cepmed approaches its fifth year of operations, we believe that Manon's background and experience is perfectly suited to take Cepmed to the next level. Going forward, we expect to increasingly attract foreign investment in our centre for translational studies, commercialize the results of our studies and implement personalized medicine tests and strategies into our healthcare system. We welcome Manon to her new position and are confident that she can take on this challenge." - Mr. Michael Saucier, Chairman of the Board, CepMed

The Board extends its best wishes to Clarissa in her new endeavors and its deep gratitude for her contributions to CepMed and the advancement of personalized medicine in Canada. "Her leadership of our public-private partnerships and innovative knowledge translation activities including a Personalized Medicine Portal and the creation of physician's expert panels in cardiology, oncology and family medicine have created an awareness of personalized medicine and its benefits across the country." - Dr. Jean Claude Tardif, Director Montreal Heart Institute Research Centre

About CepMed

Founded by the Montreal Heart Institute and Genome Quebec in 2008, CepMed is a non-profit organization dedicated to promoting the science and practice of personalized medicine. It is a Centre of Excellence for Commercialization and Research (CECR) funded by the Canadian Government, Genome Quebec and industry partners including Pfizer, AstraZeneca, Novartis and Merck. Cepmed's initiatives are designed to promote and enable personalized medical treatment, based on the use of molecular information in clinical decision making, in particular to improve the efficacy and safety of drug therapy. Cepmed participates in several multi-million dollar public-private partnerships (PPP) in translational medicine that incorporate pharmacogenomics into Phase III clinical trials or studies of marketed drugs with partners including Roche and Servier. These partnerships leverage the expertise and capacities of The Beaulieu Saucier Pharmacogenics (PGx) Centre, The Montreal Heart Institute Coordinating Centre (MHICC) and The Montreal Heart Institute Biobank. Generating several millions of dollars in investment these partnerships have established a unique and world class centre for translational clinical research. In addition CepMed is collaborating and partnering with Canadian stakeholders in personalized medicine including physicians, patients, governments, industry and investors to enable the clinical development and implementation of personalized medicine while creating commercial opportunities for Canadian companies.

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CepMed Appoints New CEO

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From Cloning ‘Dolly the Sheep’ to Curing Blindness, Scotland is on the Forefront of Life Science Discoveries

EDINBURGH, Scotland, June 18, 2012 /PRNewswire/ --A revolution in modern medicine is quietly under way in Scotland, which is rapidly emerging as a global leader in regenerative medicine and drug discovery.

Ranked #1 in the world for stem cell research, Scotland recently launched a new stem cell trial to cure corneal blindness, which could result in the development of the first harvest stem cells that restore the sight of millions of people. The revolutionary research, conducted by Advanced Cell Technologies at the Aberdeen Royal Infirmary, is the first trial of its kind ever to be carried out in the UK.

Scotland is also responsible for many other groundbreaking life science discoveries, including MRI and CAT scanners, the discovery of p53 cancer suppressor gene, world-recognized research in diabetes and cancer, ReNeuron's stem cell trial for stroke patients, and the cloning of "Dolly" the sheep.

More than two dozen Scottish life science companies and research organizations will come together to showcase these discoveries among other recent life science developments at the 2012 BIO International Convention on June 18-21 in Boston.

"Scotland may be small in size, but we're big in bioscience," said Danny Cusick, President, Americas, of Scottish Development International. "Scotland is home to some of the world's leading life science companies and has the largest concentration of animal science-related expertise and more medical research per capita than any other country in Europe."

The University of Dundee and the University of St. Andrews are both ranked among the top 10 best international academic institutions for scientists. Little wonder that the University of Dundee and the Medical Research Council just announced more than $21 million in funding from a consortium of six of the world's leading pharmaceutical companies for continuing research on the development of new drug treatments of major global diseases.

Beyond the universities, Scotland is also investing heavily in infrastructure to support development of its life science sector. Case in point is the expansive new Edinburgh BioQuarter (EBQ), which just celebrated the opening of pioneering bio-medical facilities: The Scottish Centre for Regenerative Medicine and new bio-incubator building, Nine. The EBQ was designed to foster collaboration between Scottish researchers and global life science companies that is conducive to developing and commercializing new medical discoveries.

Likewise, a former Merck research facility in Scotland's Central Belt between Glasgow and Edinburgh, is being transformed into "BioCity Scotland" to foster the growth of life science and pharmaceutical companies.

Scottish companies are also beginning to attract the notice of venture capitalists and angel funds. Boston-based Morningside Ventures, for example, recently supported Scotland-based NuCana BioMed with Series A funding.

Scotland's medical research expertise is also earning recognition on the West Coast as the country signed a Memorandum of Understanding with the prestigious San Francisco-based Californian Institute of Regenerative Medicine earlier this year, which will enable joint research and collaboration between scientists and companies in Scotland and California.

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From Cloning 'Dolly the Sheep' to Curing Blindness, Scotland is on the Forefront of Life Science Discoveries

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Eastday-Rare jail move to save a son

A FAMILY is waiting anxiously to see whether a bone marrow transplant, made possible by a rare prison transfer, has saved a boy's life.

Gao Yong, who began a 10-year sentence for burglary in 2005, was allowed to travel to donate bone marrow stem cells for his nine-year-old son, Jun Jie, who has leukemia.

Gao, who had been serving his sentence in east China's Zhejiang Province, was transferred to a prison in southwest Guizhou Province to be closer to the Xinqiao Hospital in Chongqing where Jun Ji had been taken after all possible treatments in his hometown of Zunyi in Guizhou had been exhausted.

Jun Jie was diagnosed with leukemia around the end of 2011.

Doctors at Xinqiao said Jun Jie required a bone marrow transplant, but tests showed none of his other family members were a match. His only hope was his father.

In February, after a blood sample was sent to the jail holding Gao some two hours away by air, good news came back - they matched.

Too weak to travel

"At that time, Jun Jie had become too weak to travel, so I went to judicial departments both in Zhejiang and Guizhou to persuade them to transfer his father to the Xinqiao Hospital,'' his mother Luo Jing said.

In March, Gao was transferred to the prison in Guizhou to prepare for the operation. On June 9, 10 officers escorted Gao to Chongqing.

It is very rare for a prisoner to come out of their assigned jail for as long as a week, noted the head of the escort team.

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Eastday-Rare jail move to save a son

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Stem Cell Therapy for CMT – Gary B-part 1 – Video

17-06-2012 02:12 Gary B. Stem Cell Therapy for CMT - For more info. visit

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Sistemic to Moderate Regenerative Medicine Panel at 2012 Bio International Convention

BOSTON--(BUSINESS WIRE)--

Sistemic Ltd., a leading provider of microRNA-based problem-solving services and kit-based products to the Cell Therapy community, announced today that chairman and CEO Jim Reid is moderating a panel discussion at the 2012 Bio International Convention on Wednesday, June 20, in Boston. Featuring leaders from the regenerative medicine space, the panel is titled Stem Cell Therapies Fact or Fiction, and will share the lessons learned to-date from Scottish, European and American perspectives on the path to successfulcommercialization of stem cell therapies.

Jim Reid, Sistemic CEO, commented, "Sistemic is very active in the CellTherapyarena and aremembersof Alliance for Advanced Therapies (AAT) and the Alliance for Regenerative Medicine (ARM). We see the ability to raise this topic at the leading world event, BIO 2012, as animportantstep on the path to commercialization of these products which will be transformational in healthcare, and bring hope and cures to many people around the globe."

More information on the panel at BIO 2012:

What: Panel Discussion Featuring Leaders in the Regenerative Medicine Space

When: Wednesday, June 20, 3:00PM EDT

Where: Boston Convention Center, Room 254A

Who: Leaders of the Regenerative Medicine space:

Panel objectives include evaluating lessons learned and best practice including from the Scotland Roadmap for the commercialization of stem cell therapies; identifying global (US and EU) examples of progress in stem cell therapy commercialization; and facilitating a debate on the need for a global, multi-disciplinary approach to successful commercialization of stem cell therapies.

About Sistemic Ltd

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Real-time gene sequencing used to fight MRSA

LONDON Scientists have used genome sequencing technology to control an outbreak of the superbug MRSA in a study that could point to faster and more efficient treatment of a range of diseases.

The work adds to a burgeoning body of research into better techniques for diagnosing disease more quickly and at an earlier stage to allow more effective treatment and reduce health care costs.

Much of this is being driven by whole genome sequencing, which has enabled scientists to identify the genetic markers for a range of afflictions.

MRSA, or Methicillin-Resistant Staphylococcus Aureus, is a drug-resistant bacterial infection, or superbug, and major public health problem. When outbreaks occur in hospitals it can lead to the closure of whole wards and lengthy investigations.

The bug kills an estimated 19,000 people in the United States alone each year, and even when the infection is successfully treated it can double the average length of a hospital stay and thereby increase health care costs.

A team of scientists from the Wellcome Trust Sanger Institute, the University of Cambridge and genome sequencing company Illumina Inc, used samples from a 2009 MRSA outbreak in a hospital neo-natal intensive care ward to recreate and respond to it, as if in real time.

They found that genome sequencing produced results in roughly 24 hours, using the latest technology from Illumina, gave much more detailed information.

The researchers were able to identify the particular strain of MRSA causing the outbreak, and which strains were not, quickly enough to feed back into treatment and nip the outbreak in the bud faster than current clinical testing methods.

"I think we are at the very beginning of an explosion of evidence to support the use of whole genome sequencing in public health," Sharon Peacock of Cambridge University, who led the study, told Reuters.

The research, published in the New England Journal of Medicine, comes hot on the heels of similar work done on MRSA and Clostridium difficile by a team from Oxford University with Illumina and a group of hospitals in Britain.

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Control gene for 'conveyor belt' cells could help improve oral vaccines, treat intestinal disease

Public release date: 17-Jun-2012 [ | E-mail | Share ]

Contact: Quinn Eastman qeastma@emory.edu 404-727-7829 Emory University

Scientists have found a master regulator gene needed for the development of M cells, a mysterious type of intestinal cell involved in initiating immune responses.

M cells act like "conveyor belts," ingesting bacteria and transporting substances from the gut into Peyer's patches, specialized tissues resembling lymph nodes in the intestines. Better knowledge of M cells' properties could aid research on oral vaccines and inflammatory bowel diseases.

A team of researchers at Emory University School of Medicine and RIKEN Research Center for Allergy and Immunology in Japan has identified the gene Spi-B as responsible for the differentiation of M cells.

The results are published Sunday, June 17 in the journal Nature Immunology.

"This discovery could really unlock a lot of information about the sequence of events needed for M cells to develop and what makes them distinctive," says co-author Ifor Williams, MD, PhD, associate professor of pathology and laboratory medicine at Emory University School of Medicine. "M cells have been difficult to study because they are relatively rare, they are only found within the Peyer's patches and can't be grown in isolation."

Scientists at RIKEN, led by senior author Hiroshi Ohno, MD, PhD, teamed up with Williams' laboratory, taking advantage of a discovery by Williams that a protein called RANKL, which is produced by cells in Peyer's patches, can induce M cell differentiation. Research scientist Takashi Kanaya is first author of the paper.

Kanaya and colleagues found that the gene Spi-B is turned on strongly at early stages of M cell differentiation induced by RANKL. Their suspicion of Spi-B's critical role was confirmed when they discovered that mice lacking Spi-B do not have functional M cells, and the cells in the intestines lack several other markers usually found on M cells.

"It was somewhat surprising to find Spi-B expressed in intestinal epithelial cells," Williams says. "Because Spi-B is known to be important for the development of some types of immune cells, it was thought to be expressed only in bone marrow-derived cells."

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New cerebellar ataxia gene identified in dogs

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

Contact: Professor Hannes Lohi hannes.lohi@helsinki.fi 358-919-25085 University of Helsinki

Researchers at the University of Helsinki and the Folkhlsan Research Center, Finland, have identified the genetic cause of early-onset progressive cerebellar degeneration the Finnish Hound dog breed. The study, led by Professor Hannes Lohi, revealed a new disease mechanism in cerebellar degeneration. A mutation was identified in the SEL1L gene, which has no previous link to inherited cerebellar ataxias.

This gene find is the first in canine early-onset cerebellar degeneration, and has enabled the development of a genetic test to help eradicate the disease from the breed. At the same time, SEL1L represents a novel candidate gene in human early-onset degenerative ataxias.

The research was published in the scientific journal PLoS Genetics on June 14, 2012.

Inherited ataxias affect both humans and animals. In humans, the hereditary ataxias are a heterogeneous disease group, characterized by cerebellar degeneration and dysfunction. The cerebellum is a part of the brain that is involved in coordination of movement. Degeneration of the cerebellar structures causes ataxia, which is a neurological sign of defective motor coordination that can affect gait, balance, speech and gaze. Approximately 20 known disease-causing genes have been identified in both autosomal recessive and dominant ataxias in humans but the genetic background of canine cerebellar ataxias has remained largely unknown.

The clinical signs of Finnish Hound cerebellar ataxia are present by the age of two months. The affected puppies have difficulty in controlling their leg movements and keeping their balance. The disease progresses rapidly, and in the end eating becomes impossible because of uncontrolled head movements. There is no cure for the disease and affected puppies have to be euthanized.

The research conducted by Professor Lohi and co-workers revealed marked neuronal loss in the cerebellar cortex of affected Finnish Hound puppies. By comparing the genomes of affected and healthy dogs, the cause of the disease was pinpointed to a single nucleotide change in the SEL1L gene. The nucleotide alteration causes an amino acid change in the encoded SEL1L protein.

"The identified ataxia gene is the first early-onset degenerative cerebellar ataxia gene that has been identified in dogs", says professor Hannes Lohi. In addition to Finnish Hounds, cerebellar degeneration has been identified in several other dog breeds.

"The SEL1L gene has not been previously connected to cerebellar ataxias in any species and it represents a novel candidate gene for human early-onset ataxias", Lohi continues. "In fact, we have already screened a small cohort of human patients for possible disease-causing SEL1L mutations".

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New cerebellar ataxia gene identified in dogs

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Why Genetically Engineered Food Is Dangerous

Why Genetically Engineered Food Is Dangerous

New report by genetic engineers Press release for immediate release Earth Open Source 17 June 2012

LONDON, UK - Aren't critics of genetically engineered food anti-science? Isn't the debate over GMOs (genetically modified organisms) a spat between emotional but ignorant activists on one hand and rational GM-supporting scientists on the other?

A new report released today, "GMO Myths and Truths",[1] challenges these claims. The report presents a large body of peer-reviewed scientific and other authoritative evidence of the hazards to health and the environment posed by genetically engineered crops and organisms (GMOs).

Unusually, the initiative for the report came not from campaigners but from two genetic engineers who believe there are good scientific reasons to be wary of GM foods and crops.

One of the report's authors, Dr Michael Antoniou of King's College London School of Medicine in the UK, uses genetic engineering for medical applications but warns against its use in developing crops for human food and animal feed.

Dr Antoniou said: "GM crops are promoted on the basis of ambitious claims - that they are safe to eat, environmentally beneficial, increase yields, reduce reliance on pesticides, and can help solve world hunger.

"I felt what was needed was a collation of the evidence that addresses the technology from a scientific point of view.

"Research studies show that genetically modified crops have harmful effects on laboratory animals in feeding trials and on the environment during cultivation. They have increased the use of pesticides and have failed to increase yields. Our report concludes that there are safer and more effective alternatives to meeting the world's food needs."

Another author of the report, Dr John Fagan, is a former genetic engineer who in 1994 returned to the National Institutes of Health $614,000 in grant money owing to concerns about the safety and ethics of the technology. He subsequently founded a GMO testing company.

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Life Technologies Launches Expanded, Next Generation GeneArt® Kits for Mutagenesis, Cloning and Assembly

CARLSBAD, Calif., June 18, 2012 /PRNewswire/ -- Life Technologies Corporation (LIFE) today announced the launch of three next generation GeneArt genetic engineering kits that allow molecular and synthetic biologists unprecedented speed, flexibility, precision, and efficiency for the seamless cloning, assembly, and editing of genetic material.

The GeneArt Seamless PLUS Cloning and Assembly Kit allows investigators to complete complex assembly projects in days that would take weeks with typically available methods. The kits use a proprietary enzyme mix to recognize and precisely assemble DNA fragments without the need for restriction digestion, ligation, or introduction of extra DNA sequence (seamless). The kits are designed to work with any vector a researcher chooses plus 1 to 4 fragments (or more depending on fragment sizes and workflow) in an in vitro, typically <30 minute room temperature reaction to create constructs up to 40kb in size.

The newly introduced GeneArt Seamless Cloning and Assembly Enzyme Mix is the economical choice for creating constructs up to 13kb with the option for high-throughput assembly.

The GeneArt Site-Directed Mutagenesis PLUS System can be employed to introduce deletions, insertions and substitutions ranging from small to large fragment sizes and can facilitate single or multi-site mutagenesis. Up to three sites can be edited in a single plasmid at greater than 90% efficiency. Up to 25 nucleotides can be altered in a single site, and results are delivered typically in less than three hours for plasmids up to 14 kb in size.

"Typically, mutagenesis efficiency decreases as multiple sites are targeted," said Nathan Wood, general manager and vice president of synthetic biology at Life Technologies. "The GeneArt kits maintain high efficiency over multiple sites, an advantage for our customers."

"Our scientists assemble and manipulate genetic material on a daily basis," said Wood. "They are deeply connected to the need for speed and reliability in cloning, assembly and mutagenesis systems, and they also understand that our customers need systems designed to handle different levels of complexity."

The new kits extend Life's current market-leading product offerings in assembly and mutagenesis kits, the GeneArt Seamless Cloning and Assembly Kit (for up to four fragments and constructs up to13kb), the GeneArt High-Order Genetic Assembly Kit (a yeast-based system for up to 10 fragments and constructs up to 110kb), and the GeneArt Site-Directed Mutagenesis System. With the new all-in-one enzyme/buffer mix and increased room temperature stability, the new kits offer increased flexibility, speed, and the option for high-throughput workflows. The new Mutagenesis PLUS system offers all of the single-site functionality of the current mutagenesis kits but with multi-site mutagenesis, the all-in-one enzyme/buffer mix, and increased room-temperature stability.

All products include access to Life Technologies' free online GeneArt Primer and Construct Design Tool for Seamless or High-Order Assembly and Mutagenesis: http://bioinfo.invitrogen.com/oligoDesigner/.

This tool facilitates the in silico design, assembly, or mutagenesis of a DNA molecule using GeneArt technology. The GeneArt Design Tool:

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Life Technologies Launches Expanded, Next Generation GeneArt® Kits for Mutagenesis, Cloning and Assembly

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Genetically-engineered mosquitoes can’t transmit malaria

Scientists at the University of California at Irvine and and the Pasteur Institute in Paris say theyve used genetic engineering to create mosquitoes that cant infect people with malaria. They used Anopheles stephensi mosquito a major source of malaria in India and the Middle East but say the technique could be used on dozens of different types of mosquitoes. Malaria parasites picked up by these mosquitoes are killed by the the mosquitoes immune systems. So the insects cant transmit malaria through their bites. The scientists made their announcement on June 17, 2012, and their paper was published in the Proceedings for the National Academy of Sciences.

Scientists have genetically altered the Anopheles stephensi mosquito so that their immunes systems kill the malaria parasite. They say their technique could be used with dozens of different types of mosquitoes.

More than 40 percent of the worlds population lives in areas where there is a risk of contracting malaria. The World Health Organization says there were about 216 million cases of malaria and an estimated 655,000 deaths in 2010. The deaths are largely infants, young children and pregnant women. Most deaths occur among children living in Africa where a child dies every minute from malaria.

Anthony James of UC Irvine said:

Our group has made significant advances with the creation of transgenic mosquitoes But this is the first model of a malaria vector with a genetic modification that can potentially exist in wild populations and be transferred through generations without affecting their fitness.

I did not talk to these scientists, and I have questions. What happens to the mosquitoes already in the wild, which carry the malaria parasite? Do they breed with the genetically modified mosquitoes so that some inherit malaria-killing immune systems? There will be another question for some. Is it wise to release genetically modified mosquitoes into the wild? For the families of children who might die of malaria, the answer is clear: pursue this promising line of research. The rest of us will need to acknowledge that we live in a world where the questions themselves are getting tougher.

Bottom line: Scientists at the University of California at Irvine and and the Pasteur Institute in Paris have used genetic engineering to create mosquitoes whose immune systems kill the malaria parasite. These mosquitoes, then, cant transmit malaria.

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Genetically-engineered mosquitoes can’t transmit malaria

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Genetics Meeting Surveys the Cancer Genome Landscape

Newswise BETHESDA, MD June 18, 2012 -- In cancer, the genome is shot to hell," Columbia University cell biologist I. Bernard Weinstein, M.D., famously said in 1989. Since then, researchers have catalogued the mutations that drive many human cancers. But since cancer takes years to develop, experiments on shorter-lived species have been critical in developing new diagnostics and therapeutics. Scientists who work on human cancer and those who use other species as stand-ins for humans will get together June 17-20, 2012 at the Genetics Society of Americas (GSAs) Model Organism to Human Biology (MOHB): Cancer Genetics Meeting at the Omni Shoreham Hotel in Washington, D.C.

Unlike a single-gene disease inherited through either sperm or egg, the genetic changes of cancer strike somatic (from the Greek soma meaning body) cells including those cells that make up internal organs. In affected organs, these somatic cells may have an underlying susceptibility mutation present. Once a cancer begins, an oncogene, a gene that has the potential of causing cancer, is turned on or a tumor suppressor turned off and other changes ensue. The changing nature of cancer explains why treating the disease requires staying steps ahead.

Animal models have been instrumental for working out the pathways through which all human solid tumors form. Current knowledge of cancer genes is a tribute to the basic research that has been performed over the past four decades, the majority of it in model systems, Bert Vogelstein, M.D., director, Ludwig Center at Johns Hopkins University and Investigator, Howard Hughes Medical Institute said. Dr. Vogelstein, a keynote speaker at the MOHB: Cancer Genetics Meeting has identified the sequence of genetic changes behind colorectal cancer.

Looking at the big picture, Eric Green, M.D., Ph.D., director of the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH) and another speaker at the MOHB: Cancer Genetics meeting said, Cancer is a disease of the genome. The more knowledge we gain about the structure and function of genomes, the more we will be able to learn about the genomic changes responsible for different types of cancer.

The cancer-genome connection is why NHGRI began partnering with the National Cancer Institute in 2005 to create The Cancer Genome Atlas, which will describe the genomes of 20 cancer types.

Quite a varied group of organisms have taught us about human cancers, explained Phil Hieter, Ph.D., (University of British Columbia), President of the GSA, Each model organism has its own advantages and disadvantages for the study of a particular process. The aggregate is much more powerful, so it makes great sense to shuttle among species in studying the mechanisms and mutations associated with cancer. Thats what this conference is all about.

Thanks to evolution, the cancers of model organisms reflect derangement in many of the same genes and pathways that fuel human cancers. The model organism Encyclopedia of DNA Elements (modENCODE) project, begun in 2009, is identifying the genetic controls of two popular model organisms: the roundworm Caenhorhabditis elegans and the fruit fly Drosophila melanogaster. ModENCODE has greatly advanced our knowledge of genome function in model systems, which is foundational knowledge for deciphering the biological consequences of cancer-associated genomic changes, said Dr. Green, who will discuss it at the meeting.

The mini-modENCODE Symposium being held at GSAs MOHB: Cancer Genetics Meeting will be followed by a symposium on June 20-21 hosted by NHGRI at the NIH campus to celebrate the projects accomplishments as it draws to completion this year. For more information about the NHGRI symposium, please see http://www.genome.gov/27548680.

ABOUT THE MODEL ORGANISM TO HUMAN BIOLOGY MEETING: The GSA MOHB Meeting has been held every other year since 2006. The GSA Board of Directors developed this meeting to enable basic research scientists studying genetic diseases in model organisms and scientists studying these diseases in humans to have a forum for discussion of their findings and to forge collaborative investigations.

ABOUT GSA: Founded in 1931, the Genetics Society of America (GSA) is the professional membership organization for scientific researchers, educators, bioengineers, bioinformaticians and others interested in the field of genetics. Its nearly 5,000 members work to advance knowledge in the basic mechanisms of inheritance, from the molecular to the population level. GSA is dedicated to promoting research in genetics and to facilitating communication among geneticists worldwide through its conferences, including the biennial conference on Model Organisms to Human Biology, an interdisciplinary meeting on current and cutting edge topics in genetics research, as well as annual and biennial meetings that focus on the genetics of particular organisms, including C. elegans, Drosophila, fungi, mice, yeast, and zebrafish. GSA publishes GENETICS, a leading journal in the field and an online, open-access journal, G3: Genes|Genomes|Genetics. For more information about GSA, please visit http://www.genetics-gsa.org. Also follow GSA on Facebook at facebook.com/GeneticsGSA and on Twitter @GeneticsGSA.

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Genetics Meeting Surveys the Cancer Genome Landscape

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Anti-cocaine vaccine described in Human Gene Therapy Journal

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

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, June 18, 2012A single-dose vaccine capable of providing immunity against the effects of cocaine offers a novel and groundbreaking strategy for treating cocaine addiction is described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

"This is a very novel approach for addressing the huge medical problem of cocaine addiction," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

In the article "AAVrh.10-Mediated Expression of an Anti-Cocaine Antibody Mediates Persistent Passive Immunization That Suppresses Cocaine-Induced Behavior," (http://online.liebertpub.com/doi/pdfplus/10.1089/hum.2011.178) a team of researchers from Weill Cornell Medical College (New York, NY), The Scripps Research Institute (La Jolla, CA), and Cornell University (Ithaca, NY) used a virus-based delivery vehicle in mice to transfer a gene that produces a protein capable of binding to cocaine present in the blood, preventing the cocaine from crossing into the brain. The protein is a monoclonal antibody that sequesters cocaine, making the vaccinated mice resistant to the drug's effects. Whereas unvaccinated mice exhibited hyperactivity when exposed to intravenous cocaine, the immunized mice showed no effects, according to authors Jonathan Rosenberg, et al.

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About the Journal

Human Gene Therapy (http://www.liebertpub.com/hum), the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies is an authoritative peer-reviewed journal published monthly in print and online that presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Tables of contents and a free sample issue may be viewed online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

About the Publisher

Mary Ann Liebert, Inc. (http://www.liebertpub.com) is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available at the Mary Ann Liebert, Inc. website (http://www.liebertpub.com).

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Anti-cocaine vaccine described in Human Gene Therapy Journal

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uniQure Extends Collaboration with Protein Sciences Corporation on Use of its expresSF+® Cell Line for Gene Therapy

AMSTERDAM, The Netherlands, June 18, 2012 /PRNewswire/ --

uniQure, a leader in the field of human gene therapy, announced today the extension of its collaboration with Protein Sciences Corporation ("Protein Sciences") for the exclusive use of Protein Sciences' expresSF+ (SF+) insect cell line in uniQure's AAV gene therapy programs for three specific disease indications. uniQure has the option to extend this exclusivity further into additional indications in the future.

"This agreement strengthens uniQure's gene therapy platform and further demonstrates the quality of the cell line developed by Protein Sciences." said Joern Aldag, Chief Executive of uniQure. "Protein Sciences' cell line license agreement with Merck made earlier this year and the anticipated approval of its influenza vaccine FluBlok implies SF+ technology will hold a prominent place in the manufacture of biologics."

The SF+ cell line developed by Protein Sciences is an integral component of uniQure's validated, world leading manufacturing platform. uniQure believes that this platform is the only commercially-scalable platform available for manufacturing AAV gene therapy products.

Financial terms were not disclosed.

About uniQure

uniQure is a world leader in the development ofhuman gene based therapies.uniQure has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform.This proprietary platform can be applied to a large number of rare(orphan) diseases caused by one faulty gene and allows uniQure to pursue its strategy of focusing on this sector of the industry. Further information can be found at http://www.uniqure.com.

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uniQure Extends Collaboration with Protein Sciences Corporation on Use of its expresSF+® Cell Line for Gene Therapy

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From Cloning ‘Dolly the Sheep’ to Curing Blindness, Scotland is on the Forefront of Life Science Discoveries

EDINBURGH, Scotland, June 18, 2012 /PRNewswire/ --A revolution in modern medicine is quietly under way in Scotland, which is rapidly emerging as a global leader in regenerative medicine and drug discovery.

Ranked #1 in the world for stem cell research, Scotland recently launched a new stem cell trial to cure corneal blindness, which could result in the development of the first harvest stem cells that restore the sight of millions of people. The revolutionary research, conducted by Advanced Cell Technologies at the Aberdeen Royal Infirmary, is the first trial of its kind ever to be carried out in the UK.

Scotland is also responsible for many other groundbreaking life science discoveries, including MRI and CAT scanners, the discovery of p53 cancer suppressor gene, world-recognized research in diabetes and cancer, ReNeuron's stem cell trial for stroke patients, and the cloning of "Dolly" the sheep.

More than two dozen Scottish life science companies and research organizations will come together to showcase these discoveries among other recent life science developments at the 2012 BIO International Convention on June 18-21 in Boston.

"Scotland may be small in size, but we're big in bioscience," said Danny Cusick, President, Americas, of Scottish Development International. "Scotland is home to some of the world's leading life science companies and has the largest concentration of animal science-related expertise and more medical research per capita than any other country in Europe."

The University of Dundee and the University of St. Andrews are both ranked among the top 10 best international academic institutions for scientists. Little wonder that the University of Dundee and the Medical Research Council just announced more than $21 million in funding from a consortium of six of the world's leading pharmaceutical companies for continuing research on the development of new drug treatments of major global diseases.

Beyond the universities, Scotland is also investing heavily in infrastructure to support development of its life science sector. Case in point is the expansive new Edinburgh BioQuarter (EBQ), which just celebrated the opening of pioneering bio-medical facilities: The Scottish Centre for Regenerative Medicine and new bio-incubator building, Nine. The EBQ was designed to foster collaboration between Scottish researchers and global life science companies that is conducive to developing and commercializing new medical discoveries.

Likewise, a former Merck research facility in Scotland's Central Belt between Glasgow and Edinburgh, is being transformed into "BioCity Scotland" to foster the growth of life science and pharmaceutical companies.

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From Cloning 'Dolly the Sheep' to Curing Blindness, Scotland is on the Forefront of Life Science Discoveries

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Stem Cell Therapy for CMT – Gary B-part 1 – Video

17-06-2012 02:12 Gary B. Stem Cell Therapy for CMT - For more info. visit

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Stem Cell Therapy for CMT - Gary B-part 1 - Video

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Sistemic to Moderate Regenerative Medicine Panel at 2012 Bio International Convention

BOSTON--(BUSINESS WIRE)--

Sistemic Ltd., a leading provider of microRNA-based problem-solving services and kit-based products to the Cell Therapy community, announced today that chairman and CEO Jim Reid is moderating a panel discussion at the 2012 Bio International Convention on Wednesday, June 20, in Boston. Featuring leaders from the regenerative medicine space, the panel is titled Stem Cell Therapies Fact or Fiction, and will share the lessons learned to-date from Scottish, European and American perspectives on the path to successfulcommercialization of stem cell therapies.

Jim Reid, Sistemic CEO, commented, "Sistemic is very active in the CellTherapyarena and aremembersof Alliance for Advanced Therapies (AAT) and the Alliance for Regenerative Medicine (ARM). We see the ability to raise this topic at the leading world event, BIO 2012, as animportantstep on the path to commercialization of these products which will be transformational in healthcare, and bring hope and cures to many people around the globe."

More information on the panel at BIO 2012:

What: Panel Discussion Featuring Leaders in the Regenerative Medicine Space

When: Wednesday, June 20, 3:00PM EDT

Where: Boston Convention Center, Room 254A

Who: Leaders of the Regenerative Medicine space:

Panel objectives include evaluating lessons learned and best practice including from the Scotland Roadmap for the commercialization of stem cell therapies; identifying global (US and EU) examples of progress in stem cell therapy commercialization; and facilitating a debate on the need for a global, multi-disciplinary approach to successful commercialization of stem cell therapies.

About Sistemic Ltd

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Eastday-Rare jail move to save a son

A FAMILY is waiting anxiously to see whether a bone marrow transplant, made possible by a rare prison transfer, has saved a boy's life.

Gao Yong, who began a 10-year sentence for burglary in 2005, was allowed to travel to donate bone marrow stem cells for his nine-year-old son, Jun Jie, who has leukemia.

Gao, who had been serving his sentence in east China's Zhejiang Province, was transferred to a prison in southwest Guizhou Province to be closer to the Xinqiao Hospital in Chongqing where Jun Ji had been taken after all possible treatments in his hometown of Zunyi in Guizhou had been exhausted.

Jun Jie was diagnosed with leukemia around the end of 2011.

Doctors at Xinqiao said Jun Jie required a bone marrow transplant, but tests showed none of his other family members were a match. His only hope was his father.

In February, after a blood sample was sent to the jail holding Gao some two hours away by air, good news came back - they matched.

Too weak to travel

"At that time, Jun Jie had become too weak to travel, so I went to judicial departments both in Zhejiang and Guizhou to persuade them to transfer his father to the Xinqiao Hospital,'' his mother Luo Jing said.

In March, Gao was transferred to the prison in Guizhou to prepare for the operation. On June 9, 10 officers escorted Gao to Chongqing.

It is very rare for a prisoner to come out of their assigned jail for as long as a week, noted the head of the escort team.

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Eastday-Rare jail move to save a son

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Royal Oak Veterinarian Dr. Simon First in Michigan to Offer In-House Adult Pet Stem Cell Therapy

ROYAL OAK, Mich., June 17, 2012 (GLOBE NEWSWIRE) -- Woodside Animal Hospital announced they have added both stem cell therapy and cold laser therapy to their suite of services. These two cutting edge treatments are done entirely in-house, no third-party lab work is required. Royal Oak veterinarian Dr. John Simon is the first Michigan veterinarian to provide pets with in-house adult stem cell therapy. The stem cells are derived from the pet's fat deposits and absolutely no embryonic tissue is used.

"As a holistic veterinarian, I am committed to providing high quality, cutting-edge care that combines traditional veterinary care with advanced holistic treatments," said Dr. Simon. "Our in-house stem cell therapy and cold laser therapy procedures alleviate pain in limping dogs and promote internal healing following an injury. I also recommend these procedures for pets with osteoarthritis."

Cold laser therapy is a non-surgical approach to pain management. Holistic equine veterinarians have used the procedure for over 20 years to treat injuries and joint pain. Today, veterinarians are using cold laser therapy to provide natural pain relief for injured pets.

According to Dr. Simon, cold laser therapy works by using a low-level energy beam to penetrate just below the skin's surface. Injured cells use the laser's energy to repair cellular damage. This provides relief for pain and swelling following a soft tissue injury, such as a ligament, tendon or muscle strain.

"Cold laser therapy is a revolutionary treatment for natural pain management in animals," said the Royal Oak veterinarian. "Laser therapy allows for advanced pain management, especially for pets suffering from chronic conditions or soft tissue injuries."

Woodside Animal Hospital also provides in-house pet stem cell therapy. This treatment uses adult stem cells collected from a dog's fat deposits to promote the growth of new soft tissue and cartilage. By performing the whole procedure in the clinic, the stem cells can be harvested and re-injected on the same day.

"Our in-house pet stem cell therapy is an affordable, same-day treatment that helps dogs suffering from joint pain, osteoarthritis, soft tissue injuries and hip dysplasia," said Dr. Simon. "As pets age, it's natural that their range of movement becomes restricted. While oral joint care supplements and prescription painkillers can help, medication alone cannot restore a full range of movement. Our treatments help restore activity and movement."

In addition to cold laser therapy and stem cell therapy, Dr. Simon also provides holistic treatments for cancer in dogs, cat and dog rashes, and dietary needs. The Royal Oak practice is a full-service animal hospital with wellness care, vaccinations and surgical procedures.

Dr. Simon is active in the greater Detroit veterinary community, serving as the past president of the Oakland County Veterinary Medical Association and as a board member for the Southeastern Michigan Veterinary Medical Association (SEMVMA).

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Royal Oak Veterinarian Dr. Simon First in Michigan to Offer In-House Adult Pet Stem Cell Therapy

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