Archive for March, 2015

TALIA CARLISLE/Stuff.co.nz

HOLDING ON TO HOPE: Amy Clague needs to raise $100,000 to cover flights to Russia and a stem cell transplant which she hopes will give her a chance at a normal life.

A multiple sclerosis diagnosis was not the 20th birthday present Amy Clague was hoping for.

The Melrose nanny was celebrating with family last year when she noticed something wasn't right.

"My right side was kind of numb," Clague said.

"[The next day] I woke up and it hadn't gone away. Day three it was in my face. It had spread."

Clague had no feeling on her right side from her toes to her face when she visited Wellington Hospital's emergency department for tests.

Four possible outcomes weighed on Clague's mind as she awaited the doctor's results.

"It was going to be multiple sclerosis [MS], a brain tumour, a brain bleed or a stroke."

But Clague's mother, a neurological physiotherapist who treats MS patients, knew the answer.

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21-year-old MS sufferer: 'I feel like my life is on hold'

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Newswise Researchers at the University of California, San Diego School of Medicine have identified a gene variant that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimers disease (AD). The study, published March 12 in Cell Stem Cell, is based on experiments with cultured neurons derived from adult stem cells.

Our results suggest that certain gene variants allow us to reduce the amount of beta amyloid produced by neurons, said senior author Lawrence Goldstein, PhD, director of UC San Diego Sanford Stem Cell Clinical Center and UC San Diego Stem Cell Program. This is potentially significant for slowing the progression of Alzheimers disease. AD is the most common cause of dementia in the United States, afflicting one in nine people age 65 and older.

The genetic risk factor investigated are variants of the SORL1 gene. The gene codes for a protein that affects the processing and subsequent accumulation of beta amyloid peptides, small bits of sticky protein that build up in the spaces between neurons. These plaques are linked to neuronal death and related dementia.

Previous studies have shown that certain variants of the SORL1 gene confer some protection from AD, while other variants are associated with about a 30 percent higher likelihood of developing the disease. Approximately one-third of the U.S. adult population is believed to carry the non-protective gene variants.

The studys primary finding is that variants in the SORL1 gene may also be associated with how neurons respond to a natural compound in the brain that normally acts to protect nerve cell health. The protective compound, called BDNF, short for brain-derived neurotrophic factor, is currently being investigated as a potential therapy for a number of neurological diseases, including AD, because of its role in promoting neuronal survival.

For the study, UC San Diego researchers took skin cells from 13 people, seven of whom had AD and six of whom were healthy control subjects, and reprogrammed the skin cells into stem cells. These stem cells were coaxed to differentiate into neurons, and the neurons were cultured and then treated with BDNF.

The experiments revealed that neurons that carried disease-protective SORL1 variants responded to the therapy by reducing their baseline rate of beta amyloid peptide production by, on average, 20 percent. In contrast, the neurons carrying the risk variants of the gene, showed no change in baseline beta amyloid production.

BDNF is found in everyones brain, said first author Jessica Young, PhD, a postdoctoral fellow in the Goldstein laboratory. What we found is that if you add more BDNF to neurons that carry a genetic risk factor for the disease, the neurons dont respond. Those with the protective genetic profile do.

More here:
Boosting A Natural Protection Against Alzheimer's Disease

Hip and shoulder arthritis six months after bone marrow stem cell therapy by Harry Adelson ND
Mareen describes her outcome six months after her bone marrow stem cell treatment by Harry Adelson ND for arthritis of her hip and shoulder http://www.docereclinics.com.

By: Harry Adelson, N.D.

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Hip and shoulder arthritis six months after bone marrow stem cell therapy by Harry Adelson ND - Video

Intervention Strategies for Spinal Cord Injury Patients Video: J.J. Mowder-Tinney | MedBridge
Watch the first chapter FREE: https://www.medbridgeeducation.com/courses/details/spinal-cord-injury-practical-rehabilitation-exercises Instructor: J.J. Mowde...

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Intervention Strategies for Spinal Cord Injury Patients Video: J.J. Mowder-Tinney | MedBridge - Video

Joining Forces: Wings for Life and the Christopher Dana Reeve Foundation
Wings for Life and the Christopher Dana Reeve Foundation unite to advance a groundbreaking therapy for spinal cord injury. The study, conducted by Universi...

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Orlando, Florida (PRWEB) March 12, 2015

The Miami Stem Cell Treatment Center announces a series of free public seminars on the use of adult stem cells for various degenerative and inflammatory conditions. They will be provided by Dr. Thomas A. Gionis, Surgeon-in-Chief and Dr. Nia Smyrniotis, Medical Director and Surgeon.

The seminars will be held on Tuesday, March 17, 2015, at 12:30 pm, 2:30 pm and 4:30 pm at Seasons 52, 7700 Sand Lake Road, Orlando, FL 32819. Please RSVP at (561) 331-2999.

The Miami Stem Cell Treatment Center (Miami; Boca Raton; Orlando; The Villages, FL), along with sister affiliates, the Irvine Stem Cell Treatment Center (Irvine; Westlake Villages, CA) and the Manhattan Regenerative Medicine Medical Group (Manhattan, NY), abide by approved investigational protocols using adult adipose derived stem cells (ADSCs) which can be deployed to improve patients quality of life for a number of chronic, degenerative and inflammatory conditions and diseases. ADSCs are taken from the patients own adipose (fat) tissue (found within a cellular mixture called stromal vascular fraction (SVF)). ADSCs are exceptionally abundant in adipose tissue. The adipose tissue is obtained from the patient during a 15 minute mini-liposuction performed under local anesthesia in the doctors office. SVF is a protein-rich solution containing mononuclear cell lines (predominantly adult autologous mesenchymal stem cells), macrophage cells, endothelial cells, red blood cells, and important Growth Factors that facilitate the stem cell process and promote their activity.

ADSCs are the body's natural healing cells - they are recruited by chemical signals emitted by damaged tissues to repair and regenerate the bodys injured cells. The Miami Stem Cell Treatment Center only uses Adult Autologous Stem Cells from a persons own fat No embryonic stem cells are used; and No bone marrow stem cells are used. Current areas of study include: Emphysema, COPD, Asthma, Heart Failure, Heart Attack, Parkinsons Disease, Stroke, Traumatic Brain Injury, Lou Gehrigs Disease, Multiple Sclerosis, Lupus, Rheumatoid Arthritis, Crohns Disease, Muscular Dystrophy, Inflammatory Myopathies, and degenerative orthopedic joint conditions (Knee, Shoulder, Hip, Spine). For more information, or if someone thinks they may be a candidate for one of the adult stem cell protocols offered by the Miami Stem Cell Treatment Center, they may contact Dr. Gionis or Dr. Smyrniotis directly at (561) 331-2999, or see a complete list of the Centers study areas at: http://www.MiamiStemCellsUSA.com.

About the Miami Stem Cell Treatment Center: The Miami Stem Cell Treatment Center, along with sister affiliates, the Irvine Stem Cell Treatment Center and the Manhattan Regenerative Medicine Medical Group, is an affiliate of the California Stem Cell Treatment Center / Cell Surgical Network (CSN); we are located in Boca Raton, Orlando, Miami and The Villages, Florida. We provide care for people suffering from diseases that may be alleviated by access to adult stem cell based regenerative treatment. We utilize a fat transfer surgical technology to isolate and implant the patients own stem cells from a small quantity of fat harvested by a mini-liposuction on the same day. The investigational protocols utilized by the Miami Stem Cell Treatment Center have been reviewed and approved by an IRB (Institutional Review Board) which is registered with the U.S. Department of Health, Office of Human Research Protection (OHRP); and our studies are registered with Clinicaltrials.gov, a service of the U.S. National Institutes of Health (NIH). For more information, visit our websites: http://www.MiamiStemCellsUSA.com, http://www.IrvineStemCellsUSA.com , or http://www.NYStemCellsUSA.com.

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The Miami Stem Cell Treatment Center Announces Adult Stem Cell Public Seminars in Orlando, Florida

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Newswise Researchers at the University of California, San Diego School of Medicine have identified a gene variant that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimers disease (AD). The study, published March 12 in Cell Stem Cell, is based on experiments with cultured neurons derived from adult stem cells.

Our results suggest that certain gene variants allow us to reduce the amount of beta amyloid produced by neurons, said senior author Lawrence Goldstein, PhD, director of UC San Diego Sanford Stem Cell Clinical Center and UC San Diego Stem Cell Program. This is potentially significant for slowing the progression of Alzheimers disease. AD is the most common cause of dementia in the United States, afflicting one in nine people age 65 and older.

The genetic risk factor investigated are variants of the SORL1 gene. The gene codes for a protein that affects the processing and subsequent accumulation of beta amyloid peptides, small bits of sticky protein that build up in the spaces between neurons. These plaques are linked to neuronal death and related dementia.

Previous studies have shown that certain variants of the SORL1 gene confer some protection from AD, while other variants are associated with about a 30 percent higher likelihood of developing the disease. Approximately one-third of the U.S. adult population is believed to carry the non-protective gene variants.

The studys primary finding is that variants in the SORL1 gene may also be associated with how neurons respond to a natural compound in the brain that normally acts to protect nerve cell health. The protective compound, called BDNF, short for brain-derived neurotrophic factor, is currently being investigated as a potential therapy for a number of neurological diseases, including AD, because of its role in promoting neuronal survival.

For the study, UC San Diego researchers took skin cells from 13 people, seven of whom had AD and six of whom were healthy control subjects, and reprogrammed the skin cells into stem cells. These stem cells were coaxed to differentiate into neurons, and the neurons were cultured and then treated with BDNF.

The experiments revealed that neurons that carried disease-protective SORL1 variants responded to the therapy by reducing their baseline rate of beta amyloid peptide production by, on average, 20 percent. In contrast, the neurons carrying the risk variants of the gene, showed no change in baseline beta amyloid production.

BDNF is found in everyones brain, said first author Jessica Young, PhD, a postdoctoral fellow in the Goldstein laboratory. What we found is that if you add more BDNF to neurons that carry a genetic risk factor for the disease, the neurons dont respond. Those with the protective genetic profile do.

Go here to read the rest:
Boosting A Natural Protection Against Alzheimer's Disease

Posted: Thursday, March 12, 2015 7:08 PM

UCLA stem-cell researchers have shown that a novel stem-cell gene therapy method could one day provide a one-time, lasting treatment for the most common inherited blood disorder in the U.S. sickle cell disease. Publishedin the journal Blood, the study outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, the gene correction method leads to the production of normal red blood cells. The study was directed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member, Dr. Donald Kohn.

People with sickle cell disease are born with a mutation in their beta-globin gene, which is responsible for delivering oxygen to the body through blood circulation. The mutation causes blood stem cellswhich are made in the bone marrowto produce distorted and rigid red blood cells that resemble a crescent or sickle shape. Consequently, the abnormally shaped red blood cells do not move smoothly through blood vessels, resulting in insufficient oxygen supply to vital organs. Anyone can be born with sickle cell disease, but it occurs more frequently in African Americans and Hispanic Americans.

The stem-cell gene therapy method described in the study seeks to directly correct the mutation in the beta-globin gene so bone marrow stem cells then produce normal, circular-shaped blood cells that do not sickle. The fascinating gene correction technique used specially engineered enzymes, called zinc-finger nucleases, tocut out the mutated genetic code and replace it with a corrected version that repairs the beta-globin mutation.

For the study, bone marrow stem cells donated by people with the sickle cell gene mutation were treated in the laboratory with the zinc-finger nucleases enzyme cutting method.Kohn and his team then demonstrated in mouse models that thecorrected bone-marrow stem cells have the capability to replicate successfully. The research showed that the method holds the potential to permanently treat the disease if a higher level of correction is achieved.

This is a very exciting result,said Dr. Kohn, professor of pediatrics atUCLAs David Geffen School of Medicine, professor of microbiology, immunology and molecular genetics in Life Sciences at UCLA, member of the UCLA Childrens Discovery and Innovation Institute at Mattel Childrens Hospital and senior author on the study. It suggests the future direction for treating genetic diseases will be by correcting the specific mutation in a patients genetic code. Since sickle cell disease was the first human genetic disease where we understood the fundamental gene defect,and since everyone with sickle cell has the exact same mutation in the beta-globin gene, it is a great target for this gene correction method.

To make the cut in the genetic code, Dr. Kohn and his team used zinc-finger nucleases engineered by Sangamo BioSciences, Inc., in Richmond. The enzymes can be designed to recognize a specific and targeted point in the genetic code. For the study, scientists at Sangamo BioSciences engineered the enzymes to create a cut at the site of the mutated genetic code that causes sickle cell disease. This break triggered a natural process of repair in the cell and at the same time, a molecule containing the correct genetic code was inserted to replace the mutated code.

The next steps in this research will involve improving the efficiency of the mutation correction process and performing pre-clinical studies to demonstrate that the method is effective and safe enough to move to clinical trials.

Symptoms of sickle cell disease usually begin in early childhood and include a low number of red blood cells (anemia), repeated infections and periodic episodes of pain. People with sickle cell disease typically have a shortened lifespan of just 36-40 years of age. The disease impacts more than 250,000 new patients worldwide each year. The only cure currently available for sickle cell disease is a transplant of bone marrow stem cells from a matched sibling, but matches are rare or can result in rejection of the transplanted cells.

This is a promising first step in showing that gene correction has the potential to help patients with sickle cell disease, said Megan Hoban, a senior graduate student in microbiology, immunology and molecular genetics and first author on the study. The study data provide the foundational evidence that the method is viable.

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UCLA Research Shows Promising Method For Correcting Genetic Code To Treat Sickle Cell Disease

The Genetic Engineering of Humans
by Taylor Davidson.

By: STS1 2015 Student Media Productions

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The Genetic Engineering of Humans - Video

IMAGE:This photo is of the sighted, surface-dwelling fish related to the ancient, eyeless Astyanax mexicanus. view more

Credit: Jay Yocis

A $1.92 million, five-year R01 Award from the National Institutes of Health will support University of Cincinnati research into the genetic aspects of craniofacial asymmetries that could address a wide spectrum of human conditions, from non-syndromic cleft palate to hemifacial microsomia - conditions that can impair breathing or lead to emotional suffering from distorted appearance. In addition, UC biology researcher Joshua Gross, an assistant professor of biological sciences, was awarded $519,343 from the National Science Foundation to explore the genetic explanation for pigmentation loss in cave animals, which could also hold links to pigmentation changes in humans. Both awards get underway in March.

The researchers are searching for genetic hints by examining a species of eyeless, cave-dwelling fish, Astyanax mexicanus - which has lived in the pitch-black caves of the Sierra de El Abra region of Mexico for millions of years. These fish can be compared with the closely related sighted surface-dwelling fish that are found in Mexico, Texas and New Mexico. Previous research suggests that genetic mutations leading to craniofacial distortions in the cavefish may be similar to human facial abnormalities that often result in painful, corrective surgeries as early as infancy. The closely-related surface-dwelling fish do not have these facial abnormalities.

The funding will support genome-wide mapping which will allow researchers to zero in on the precise region of the genome - specific genes as well as mutations within genes - that will explain these facial asymmetries.

The research project will examine these three levels:

Hello, Gorgeous - The 'Beautiful Reflection,' or Brangelina Factor

Gross says the project began with an appreciation for the fact that symmetry is an important component of human perceptions of facial attractiveness. "This trait evolves under intense sexual selection as a signal of robust physical health and genetic quality in potential mates," states the research proposal. "Think of couples like Brad Pitt and Angelina Jolie, who are admired worldwide for their physical features," says Gross. "The logical flow of this is that facial attractiveness is believed to be an indication of strong genetic composition - a strong mate who will provide for your offspring - and so indirectly there may have been evolutionary pressures acting on our ancestors to maintain facial symmetry in humans.

"Cavefish have naturally lost their eyes over the course of evolution," continues Gross. "The fish can't see one another anymore, so the left and right sides of their faces become uncoupled and begin to exhibit random asymmetries. One of our most surprising discoveries is that there's actually a genetic basis for that asymmetry. Some changes in the genome have resulted in one side of the face developing differently from the other side of the face. Because this process occurs so often, cavefish are a powerful natural model system for learning about this fundamental biological phenomenon of craniofacial symmetry."

The UC researchers have previously found two genes in the cavefish that are closely tied to non-syndromic cleft palate in humans.

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NIH awards UC biologist $1.9 million for genetic research

Case Western Reserve scientists have discovered that speed matters when it comes to how messenger RNA (mRNA) deciphers critical information within the genetic code -- the complex chain of instructions critical to sustaining life. The investigators' findings, which appear in the March 12 journal Cell, give scientists critical new information in determining how best to engage cells to treat illness -- and, ultimately, keep them from emerging in the first place.

"Our discovery is that the genetic code is more complex than we knew," said senior researcher Jeff Coller, PhD, associate professor, Division of General Medical Sciences, and associate director, The Center for RNA Molecular Biology, Case Western Reserve University School of Medicine. "With this information, researchers can manipulate the genetic code to achieve more predictable outcomes in an exquisite fashion."

The genetic code is a system of instructions embedded within DNA. The code tells a cell how to generate proteins that control cellular functions. mRNA transmits the instructions from DNA to ribosomes. Ribosomes translate the information contained within the mRNA and produce the instructed protein. The genetic code comprises 61 words, called "codons," and a single codon, a sequence of three nucleotides, instructs the ribosome how to build proteins.

The code not only dictates what amino acids are incorporated into proteins, it also tells the cell how fast they should be incorporated. With this information, researchers can manipulate the genetic code to achieve predictable protein levels in an exquisite fashion."

The most significant breakthrough in the Case Western Reserve work is that all of the words, or codons, in the genetic code are deciphered at different rates; some are deciphered rapidly while others are deciphered slowly. The speed of how mRNA decodes its information is the sum of all the codons it contains. This imposed speed limit then ultimately affects the amount of protein produced. Sometimes faster is better to express a high level of protein. Sometimes slower is better to limit the amount protein. Importantly, codons are redundant -- many of these words mean the same thing.

Coller and colleagues found that each of the codons is recognized differently by a ribosome. Some codons are recognized faster than others, but these differences in speed are tiny. Over the entire span of an mRNA, however, each tiny difference in speed is powerfully additive.

"Many codons mean the same thing, but they influence decoding rate differently. Because of this, we can change an mRNA without changing its protein sequence and cause it to be highly expressed or poorly expressed and anywhere in between," he said. "We can literally dial up or down protein levels any way we want now that we know this information."

During their research, investigators measured the mRNA decay rate for every transcript within the cell. They were seeking answers for why different RNAs had different stabilities. With statistical analysis, investigators compared the half-lives of mRNAs to the codons used within these messages. A strong correlation emerged between codon identity and mRNA message stability. They ultimately linked these observations back to the process of mRNA translation.

"mRNA translation and mRNA decay are intimately connected. This can be very beneficial to scientists. If you would like a gene to be expressed really well, you simply change the protein sequence to be derived by all optimal codons. This will both stabilize the mRNA and cause it to be translated more efficiently," Coller said. "If you need an mRNA to express at a low level, you fill it with non-optimal codons. The mRNA will be poorly translated and thus unstable. Evolution has used codon optimization to shape the expression of the proteome. Genes of similar function use similar codons; therefore, they are expressed at similar levels."

His discovery has a variety of practical implications for medicine. From a bioengineering perspective, molecular biology techniques can be applied to manipulate the gene to contain ideal codons and obtain the gene expression pattern that is most beneficial to the application. From a human physiological standpoint, it's possible to learn the speed limit for each and every mRNA and then determine if this changes in specific pathologies such as cancer. Currently, it is unknown whether codons convey different speeds in disease states. A future direction for research will be to link codon speeds to specific illnesses. The potential is also there to develop drugs that can manipulate higher or lower gene expression by changing the decoding rate.

See the original post:
Hidden meaning and 'speed limits' found within genetic code

Case Western Reserve scientists have discovered that speed matters when it comes to how messenger RNA (mRNA) deciphers critical information within the genetic code -- the complex chain of instructions critical to sustaining life. The investigators' findings, which appear in the March 12 journal Cell, give scientists critical new information in determining how best to engage cells to treat illness -- and, ultimately, keep them from emerging in the first place.

"Our discovery is that the genetic code is more complex than we knew," said senior researcher Jeff Coller, PhD, associate professor, Division of General Medical Sciences, and associate director, The Center for RNA Molecular Biology, Case Western Reserve University School of Medicine. "With this information, researchers can manipulate the genetic code to achieve more predictable outcomes in an exquisite fashion."

The genetic code is a system of instructions embedded within DNA. The code tells a cell how to generate proteins that control cellular functions. mRNA transmits the instructions from DNA to ribosomes. Ribosomes translate the information contained within the mRNA and produce the instructed protein. The genetic code comprises 61 words, called "codons," and a single codon, a sequence of three nucleotides, instructs the ribosome how to build proteins.

The code not only dictates what amino acids are incorporated into proteins, it also tells the cell how fast they should be incorporated. With this information, researchers can manipulate the genetic code to achieve predictable protein levels in an exquisite fashion."

The most significant breakthrough in the Case Western Reserve work is that all of the words, or codons, in the genetic code are deciphered at different rates; some are deciphered rapidly while others are deciphered slowly. The speed of how mRNA decodes its information is the sum of all the codons it contains. This imposed speed limit then ultimately affects the amount of protein produced. Sometimes faster is better to express a high level of protein. Sometimes slower is better to limit the amount protein. Importantly, codons are redundant -- many of these words mean the same thing.

Coller and colleagues found that each of the codons is recognized differently by a ribosome. Some codons are recognized faster than others, but these differences in speed are tiny. Over the entire span of an mRNA, however, each tiny difference in speed is powerfully additive.

"Many codons mean the same thing, but they influence decoding rate differently. Because of this, we can change an mRNA without changing its protein sequence and cause it to be highly expressed or poorly expressed and anywhere in between," he said. "We can literally dial up or down protein levels any way we want now that we know this information."

During their research, investigators measured the mRNA decay rate for every transcript within the cell. They were seeking answers for why different RNAs had different stabilities. With statistical analysis, investigators compared the half-lives of mRNAs to the codons used within these messages. A strong correlation emerged between codon identity and mRNA message stability. They ultimately linked these observations back to the process of mRNA translation.

"mRNA translation and mRNA decay are intimately connected. This can be very beneficial to scientists. If you would like a gene to be expressed really well, you simply change the protein sequence to be derived by all optimal codons. This will both stabilize the mRNA and cause it to be translated more efficiently," Coller said. "If you need an mRNA to express at a low level, you fill it with non-optimal codons. The mRNA will be poorly translated and thus unstable. Evolution has used codon optimization to shape the expression of the proteome. Genes of similar function use similar codons; therefore, they are expressed at similar levels."

His discovery has a variety of practical implications for medicine. From a bioengineering perspective, molecular biology techniques can be applied to manipulate the gene to contain ideal codons and obtain the gene expression pattern that is most beneficial to the application. From a human physiological standpoint, it's possible to learn the speed limit for each and every mRNA and then determine if this changes in specific pathologies such as cancer. Currently, it is unknown whether codons convey different speeds in disease states. A future direction for research will be to link codon speeds to specific illnesses. The potential is also there to develop drugs that can manipulate higher or lower gene expression by changing the decoding rate.

Read more:
Case Western Reserve scientists find hidden meaning and 'speed limits' within genetic code

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Newswise Case Western Reserve scientists have discovered that speed matters when it comes to how messenger RNA (mRNA) deciphers critical information within the genetic code the complex chain of instructions critical to sustaining life. The investigators findings, which appear in the March 12 journal Cell, give scientists critical new information in determining how best to engage cells to treat illness and, ultimately, keep them from emerging in the first place.

Our discovery is that the genetic code is more complex than we knew, said senior researcher Jeff Coller, PhD, associate professor, Division of General Medical Sciences, and associate director, The Center for RNA Molecular Biology, Case Western Reserve University School of Medicine. With this information, researchers can manipulate the genetic code to achieve more predictable outcomes in an exquisite fashion.

The genetic code is a system of instructions embedded within DNA. The code tells a cell how to generate proteins that control cellular functions. mRNA transmits the instructions from DNA to ribosomes. Ribosomes translate the information contained within the mRNA and produce the instructed protein. The genetic code comprises 61 words, called codons, and a single codon, a sequence of three nucleotides, instructs the ribosome how to build proteins.

The code not only dictates what amino acids are incorporated into proteins, it also tells the cell how fast they should be incorporated. With this information, researchers can manipulate the genetic code to achieve predictable protein levels in an exquisite fashion.

The most significant breakthrough in the Case Western Reserve work is that all of the words, or codons, in the genetic code are deciphered at different rates; some are deciphered rapidly while others are deciphered slowly. The speed of how mRNA decodes its information is the sum of all the codons it contains. This imposed speed limit then ultimately affects the amount of protein produced. Sometimes faster is better to express a high level of protein. Sometimes slower is better to limit the amount protein. Importantly, codons are redundant many of these words mean the same thing.

Coller and colleagues found that each of the codons is recognized differently by a ribosome. Some codons are recognized faster than others, but these differences in speed are tiny. Over the entire span of an mRNA, however, each tiny difference in speed is powerfully additive.

Many codons mean the same thing, but they influence decoding rate differently. Because of this, we can change an mRNA without changing its protein sequence and cause it to be highly expressed or poorly expressed and anywhere in between, he said. We can literally dial up or down protein levels any way we want now that we know this information.

During their research, investigators measured the mRNA decay rate for every transcript within the cell. They were seeking answers for why different RNAs had different stabilities. With statistical analysis, investigators compared the half-lives of mRNAs to the codons used within these messages. A strong correlation emerged between codon identity and mRNA message stability. They ultimately linked these observations back to the process of mRNA translation.

View post:
Case Western Reserve Scientists Discover Hidden Meaning and 'Speed Limits' within the Genetic Code

Genetics Problems Based on Mendel #39;s Laws - Sex Linkage
Learn about Mendel #39;s Laws and Sex Linkage through genetics problems, such as red-green color blindness and dimple inheritance.

By: Elon TLT

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Genetics Problems Based on Mendel's Laws - Sex Linkage - Video

Father of Genetics
Introduction to genetics and Gregor Mendel.

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LONDON: Pakistani and Bangladeshi people in London's least healthy boroughs are being asked to provide spit samples and health records to researchers hoping to find genetic clues to why they are disproportionately affected by certain diseases.

The East London Genes and Health project will focus partly on so-called knock-out genes, rare in the general population but more frequent in communities where cousins and other close relatives marry and have children, as is more common in Pakistani and Bangladeshi communities.

Read: Have Bangladeshis overtaken Pakistanis in Britain?

The largest community genetics study in the world will recruit 100,000 volunteers from East London, which have substantial South Asian populations.

Researchers leading the study say health signals buried in the data could have a big impact on peoples' health worldwide.

This is the first time a large-scale genetics study has focused on two distinct ethnic minority groups, with high levels of health concerns in the community and the potential for significant genetic variation, Richard Trembath, a professor at Queen Mary University of London, told reporters at a briefing.

These findings will play a key role in tackling health inequality locally and in the UK, (and) we hope to reveal crucial information about the link between genetics and common diseases which will have significant international impact.

Studying genetic variation is crucial to improving understanding of the normal variation in genes in certain populations, which can then help the diagnosis of inherited rare diseases.

So-called knock-out genes occur when a healthy person has two copies, inherited from both parents, of a gene that functions differently to the norm.

The team hopes to use these findings to understand how knock-out genes impact health and eventually to help develop new drugs or treatments which block bad genes and enhance good ones.

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Genetics study seeks South Asian health clues in East London - Pakistan - DAWN.COM

Gene-editing companies say research on altering the DNA of human reproductive cells is dangerous and unethical.

Officials of a biotechnology industry group have called for a voluntary moratorium on using new DNA-editing techniques to change the genetic characteristics of human embryos in laboratory research.

In an editorial published today by the journal Nature, Edward Lanphier, CEO of the biotechnology company Sangamo Biosciences, and four colleagues write that scientists should agree not to modify the DNA of human reproductive cells because it raises safety and ethical risks including the danger of unpredictable effects on future generations.

New gene-editing techniques, in particular one called CRISPR, have given scientists powerful and useful new ways to swap and change DNA letters inside of living cells for the first time (see Genome Surgery).

Recently, some scientific teams have started to study whether CRISPR would be able to correct disease genes in future generations of peoplefor instance, by repairing genes during in vitro fertilization, or in eggs or sperm. The idea of such germ line modification would be to install healthy versions of genes, which children would be born with.

The emergence of active research around germ-line editing, which is taking place in China, at Harvard University, and at a publicly traded biotechnology company called OvaScience, were described last week by MIT Technology Review (see Engineering the Perfect Baby).

But the idea of using editing technology to improve children is as controversial as it is medically powerful. In their editorial, Lanphier, whose coauthors include Fyodor Urnov, co-developer of a different gene-editing system, raise the concern that such techniques might be exploited for non-therapeutic modifications. That could mean, for instance, changing the physical traits of children.

The availability of technology to carry out genetic engineering in human germ-line cells is driving intense debate in scientific circles and may eventually become a legal issue in the United States and other countries.

The authors call for a cessation of basic research is unusual and likely to be opposed by scientists as an intrusion on the quest for scientific knowledge.

George Church, a professor at Harvard Medical School whose laboratory studies CRISPR and germ-line editing, says a voluntary moratorium would be weak compared with existing regulations that nearly all countries impose on the use of new medical technologies until they are proven safe and effective in animals or human [tests]. Church was referring to rules governing the birth of actual gene-edited children, not basic research.

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Industry Body Calls for Gene-Editing Moratorium

What can be done to make life easier for paraplegics and their loved ones? | The Cagle Law Firm
http://www.allinjuryattorney.com If you or a loved one has suffered a spinal cord injury, there has been a serious change in your lifestyle. Medical advancements and technology influence long-term...

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What can be done to make life easier for paraplegics and their loved ones? | The Cagle Law Firm - Video

WSCS 2014: DEVELOPMENT OF JAPANESE REGENERATIVE MEDICINE INDUSTRY
Presenter - Takuya Yokokawa, FujiFilm.

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WSCS 2014: DEVELOPMENT OF JAPANESE REGENERATIVE MEDICINE INDUSTRY - Video

Kansas Regenerative Medicine
2015 Emerging Existing Business Award Winner with the Kansas Small Business Development Center.

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Kansas Regenerative Medicine - Video

The growing number of biological structures being grown on chips in various laboratories around the world is rapidly replicating the entire gamut of major human organs. Now one of the most important of all a viable functioning heart has been added to that list by researchers at the University of California at Berkeley (UC Berkeley) who have taken adult stem cells and grown a lattice of pulsing human heart tissue on a silicon device.

Sourced from human-induced pluripotent stem cells able to be persuaded into forming many different types of tissue, the human heart device cells are not simply separate groups of cells existing in a petri dish, but a connected series of living cells molded into a structure that is able to beat and react just like the real thing.

"This system is not a simple cell culture where tissue is being bathed in a static bath of liquid," said study lead author Anurag Mathur, a postdoctoral researcher at UC Berkeley. "We designed this system so that it is dynamic; it replicates how tissue in our bodies actually gets exposed to nutrients and drugs."

Touted as a possible replacement for living animal hearts in drug-safety screening, the ability to easily access and rapidly analyze a heart equivalent in experiments presents appealing advantages.

"Ultimately, these chips could replace the use of animals to screen drugs for safety and efficacy," said professor of bioengineering at UC Berkeley, and leader of the research team, Kevin Healy.

The cardiac microphysiological system, as the team calls its heart-on-a-chip, has been designed so that its silicon support structure is equivalent to the arrangement and positioning of conjoining tissue filaments in a human heart. To this supporting arrangement, the researchers loaded the engineered human heart cells into the priming tube, whose cone-shaped funnel assisted in aligning the cells in a number of layers and in one direction.

In this setup, the team created microfluidic channels on each side of the cell holding region to replicate blood vessels to imitate the interchange of nutrients and drugs by diffusion in human tissue. The researchers believe that this arrangement may also one day provide the ability to view and gauge the expulsion of metabolic waste from the cells in future experiments.

"Many cardiovascular drugs target those channels, so these differences often result in inefficient and costly experiments that do not provide accurate answers about the toxicity of a drug in humans," said Professor Healy. "It takes about US$5 billion on average to develop a drug, and 60 percent of that figure comes from upfront costs in the research and development phase. Using a well-designed model of a human organ could significantly cut the cost and time of bringing a new drug to market."

The use of animal organs to forecast human reactions to new drugs is problematic, the UC Berkeley researchers note, citing the fundamental differences between species as being responsible for high failure rates in using these models. One aspect responsible for this failure is to be found in the difference in the ion channel structure between human and other animals where heart cells conduct electrical currents at different rates and intensities. It is the standardized nature of using actual human heart cells that the team sees as the heart-on-a-chip's distinct advantage over animal models.

The UC Berkeley device is certainly not the first replication of an organ-on-a-chip, but potentially one of the first successful ones to integrate living cells and artificial structures in a single functioning unit. Harvard's spleen-on-a-chip, for example, replicates the operation of the spleen, but does so by using a set of circulatory tubes containing magnetic nanobeads.

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Heart-on-a-chip beats a steady rhythm

IMAGE:The success of the new technique suggests the possibility of implanting these tweaked cells back into a patient so they can generate an immune system. Though the new work, published... view more

LA JOLLA--For infants with severe combined immunodeficiency (SCID), something as simple as a common cold or ear infection can be fatal. Born with an incomplete immune system, kids who have SCID--also known as "bubble boy" or "bubble baby" disease--can't fight off even the mildest of germs. They often have to live in sterile, isolated environments to avoid infections and, even then, most patients don't live past a year or two. This happens because stem cells in SCID patients' bone marrow have a genetic mutation that prevents them from developing critical immune cells, called T and Natural Killer (NK) cells.

Now, Salk researchers have found a way to, for the first time, convert cells from x-linked SCID patients to a stem cell-like state, fix the genetic mutation and prompt the corrected cells to successfully generate NK cells in the laboratory.

The success of the new technique suggests the possibility of implanting these tweaked cells back into a patient so they can generate an immune system. Though the new work, published March 12, 2015 in Cell Stem Cell, is preliminary, it could offer a potentially less invasive and more effective approach than current options.

"This work demonstrates a new method that could lead to a more effective and less invasive treatment for this devastating disease," says senior author Inder Verma, Salk professor and American Cancer Society Professor of Molecular Biology. "It also has the potential to lay the foundation to cure other deadly and rare blood disorders."

Previous attempts to treat SCID involved bone marrow transplants or gene therapy, with mixed results. In what began as promising clinical trials in the 1990s, researchers hijacked virus machinery to go in and deliver the needed genes to newly growing cells in the patient's bone marrow. While this gene therapy did cure the disease at first, the artificial addition of genes ended up causing leukemia in a few of the patients. Since then, other gene therapy methods have been developed, but these are generally suited for less mild forms of the disease and require bone marrow transplants, a difficult procedure to perform on critically sick infants.

To achieve the new method, the Salk team secured a sample of bone marrow from a deceased patient in Australia. Using that small sample, the team developed the new method in three steps. First, they reverted the patient cells into induced pluripotent stem cells (iPSCs)--cells that, like embryonic stem cells, have the ability to turn into any type of tissue and hold vast promise for regenerative medicine.

"Once we had patient-derived stem cells, we could remove the genetic mutation, essentially fixing the cells," explains one of the first authors and Salk postdoctoral researcher Amy Firth.

The second innovation was to use new gene editing technology to correct the SCID-related genetic deficiency in these iPSCs. To remove the mutation, the researchers used a technology called TALEN (similar to the better known CRISPR method). This set of enzymes act as molecular scissors on genes, letting researchers snip away at a gene and replace the base pairs that make up DNA with other base pairs.

"Unlike traditional gene therapy methods, we aren't putting a whole new gene into a patient, which can cause unwanted side effects," says Tushar Menon, first author and Salk postdoctoral researcher. "We use TALEN-based genome editing to change just one nucleotide in one gene to correct the deficiency. The technique is literally that precise."

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Immune system-in-a-dish offers hope for 'bubble boy' disease

March 12, 2015 // R. Colin Johnson

Living beating hearts on-a-chip were recently created from pluripotent stem cells discovered by 2010 Kyoto Prize Winner, Shinya Yamanaka.

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Bioengineers at the University of Berkeley aim to create all of the human organs on-a-chip then connect them with micro-fluidic channels to create a complete human-being on-a-wafer.

"We have learned how to derive almost any type of human tissue from skin stem cells as was first discovered by Yamanaka," professor Kevin Healy told EE Times. "Our initial application is drug screening without having to use animals, but putting organs-on-a-chip using the stem cells of the patient could help with genetic diseases as well."

"For instance, one drug might solve a heart problem, but create toxins in the liver," Healy told us. "Which would be much better to find out before administering to the patient."

As to creating living robots in this way, Healy said that was not their mission on the current project, since their funding in coming from the National Institutes of Health's (NIH's) Tissue Chip for Drug Screening Initiative, an interagency collaboration specifically aimed at developing 3-D human tissue chips for drug screening.

However, the technology being creating, especially the microfluidic channels connecting the organs-on-a-chip so that they interact, could someday serve as a basis for making robot-like creatures.

"What we would need for that is sensors and actuators. Sensors would be the easiest, but MIT in particular is working on artificial muscles to serve as actuators," Healy told us.

Living beating hearts on-a-chip were recently created from pluripotent stem cells discovered by 2010 Kyoto Prize Winner, Shinya Yamanaka.

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Heart on-a-chip beats

IMAGE:Brain tumor stem cells (orange) in mice express a stem cell marker (green). Researchers at Washington University School of Medicine in St. Louis are studying how cancer stem cells make... view more

Credit: Yi-Hsien Chen

Scientists are eager to make use of stem cells' extraordinary power to transform into nearly any kind of cell, but that ability also is cause for concern in cancer treatment. Malignant tumors contain stem cells, prompting worries among medical experts that the cells' transformative powers help cancers escape treatment.

New research proves that the threat posed by cancer stem cells is more prevalent than previously thought. Until now, stem cells had been identified only in aggressive, fast-growing tumors. But a mouse study at Washington University School of Medicine in St. Louis shows that slow-growing tumors also have treatment-resistant stem cells.

The low-grade brain cancer stem cells identified by the scientists also were less sensitive to anticancer drugs. By comparing healthy stem cells with stem cells from these brain tumors, the researchers discovered the reasons behind treatment resistance, pointing to new therapeutic strategies.

"At the very least, we're going to have to use different drugs and different, likely higher dosages to make sure we kill these tumor stem cells," said senior author David H. Gutmann, MD, PhD, the Donald O. Schnuck Family Professor of Neurology.

The research appears online March 12 in Cell Reports.

First author Yi-Hsien Chen, PhD, a senior postdoctoral research associate in Gutmann's laboratory, used a mouse model of neurofibromatosis type 1 (NF1) low-grade brain tumors to identify cancer stem cells and demonstrate that they could form tumors when transplanted into normal, cancer-free mice.

NF1 is a genetic disorder that affects about 1 in every 2,500 babies. The condition can cause an array of problems, including brain tumors, impaired vision, learning disabilities, behavioral problems, heart defects and bone deformities.

The most common brain tumor in children with NF1 is the optic glioma. Treatment for NF1-related optic gliomas often includes drugs that inhibit a cell growth pathway originally identified by Gutmann. In laboratory tests conducted as part of the new research, it took 10 times the dosage of these drugs to kill the low-grade cancer stem cells.

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Stem cells lurking in tumors can resist treatment

A new study by University of Alberta law researchers reveals sometimes overly optimistic news coverage of clinical translation of stem cell therapies--and as spokespeople, scientists need to be mindful of harnessing public expectations.

"As the dominant voice in respect to timelines for stem cell therapies, the scientists quoted in these stories need to be more aware of the importance of communicating realistic timelines to the press," said researcher Kalina Kamenova, who co-authored the study with professor Timothy Caulfield in the University of Alberta's Health Law Institute, based in the Faculty of Law.

Their analysis of media coverage showed that most news reports were highly optimistic about the future of stem cell therapies and forecasted unrealistic timelines for clinical use. The study, published in the latest issue of Science Translational Medicine, examined 307 news reports covering translational stem cell research in major daily newspapers in Canada, the United States and the United Kingdom between 2010 and 2013.

While the field of stem cell research holds tremendous promise, "it has also been surrounded by tremendous hype, and we wanted to quantify that in some degree," Caulfield said. "Pop culture representations have an impact on how the public perceives the readiness of stem cell research, and that in turn feeds into stem cell tourism, marketing of unproven therapies and even the public's trust in research. We wanted to provide findings that would help inform the issue."

Their study found that 69 per cent of all news stories citing timelines predicted that therapies would be available within five to 10 years or even sooner. At the same time, the press overlooked challenges and failures in therapy translation, such as the discontinuation of the first FDA-approved clinical trial of an embryonic stem cell-derived therapy for spinal cord injuries in 2011. The biotech company conducting the trial was a leader in embryonic stem cell therapies and its decision to stop its work on stem cells was considered a significant setback for the field.

As well, ethical concerns about the use of human embryonic stem cells were displaced from the forefront of news coverage, while the clinical translation of stem cell therapies and new discoveries, such as hockey star Gordie Howe's recent treatment, grabbed the headlines instead.

"Our findings showed that many scientists have often provided either by implication or direct quotes, authoritative statements regarding unrealistic timelines for stem cell therapies and media hype can foster unrealistic public expectations about clinical translation and increased patient demand for unproven stem cell therapies," Caulfield noted.

While stem cell therapy research is progressing and has seen a dramatic increase in the past decade of clinical trials for treatments, the vast majority of these studies are still in the safety-testing stage and involve a limited number of participants, Kamenova noted.

"The approval process for new treatments is long and complicated, and only a few of all drugs that enter pre-clinical testing are approved for human clinical trials. It takes on average 12 years to get a new drug from the lab to the market, and additional 11 to 14 years of post-market surveillance," she added.

The science world is under pressure to come up with cures for what ails us, but "care needs to be taken by the media and the research community so that advances in research and therapy are portrayed in a realistic manner," Caulfield said.

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Media portray unrealistic timelines for stem cell therapies