What is gene therapy?

Gene therapy is the use of genetic instructions to produce a protein to treat a disorder or deficiency. It can aid in a disease even if the therapy is not directly targeting a gene defect that causes the disease. In amyotrophic lateral sclerosis (ALS), gene therapy may help if it can deliver a beneficial protein, to salvage dying nerve cells. The gene therapy simply is a means to boost on site production of a trophic (growth enhancing) factor, at places where nerve cells are in trouble.

Genes are the molecules in all cells of our bodies that carry the instructions to make all of the materials that comprise the body. In the 1950s, scientists determined that genes code precisely for proteins, with a sequence that specifies the order of the building blocks of proteins, the amino acids. Each gene corresponds to a protein. Each base in a gene codes for an amino acid. The order of bases in a gene produces the ordered chain of amino acids that produce a working protein.

At the turn of the current century, scientists determined, in rough draft form, the sequence of all of the human genes. By this time, they also knew how to create a gene construct, and move that construct into cells, to get the cells to make the corresponding protein.

In some diseases, researchers already know that a defective gene is not able to work. They have the potential means to cure the disease, by replacing the defective gene with a correct, working copy. In ALS, only a few percent of patients have a known gene defect. For the rest, it may be one undiscovered gene that is the problem, or it may be several. But gene therapy can still be designed to aid patients with ALS by providing supportive proteins for nerve cells.

Vectors deliver genes Genes normally reside in the nucleus, the core of a cell, separated from the surrounding materials by a membrane. The chromosomes are the structures within the cell nucleus that contain the DNA that comprises the genes. It is very challenging to get a gene made in the lab to cross both the outer envelope of a cell, and the nuclear membrane as well, to reach the chromosomes.

Scientists studying viruses have discovered natures own solution to the problem of moving genes. Viruses are essentially genes that have evolved to hijack cells, instead of forming cells for themselves. So viruses have strategies to enter cells and take over the protein production process, to produce instead, the virus. Researchers have figured out how to use viruses as Trojan horses, to bring in genes that can then carry out genetic repairs, replacing defective DNA.

For many viruses, researchers can disarm the genes responsible for the damaging properties and put in, instead, genetic instructions to make therapeutic proteins. These viruses, redesigned by researchers, are called vectors. They are simply a means to smuggle in therapeutic genes.

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Gene Therapy - The ALS Association

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In 1872, the American physician George Huntington wrote about an illness that he called "an heirloom from generations away back in the dim past." He was not the first to describe the disorder, which has been traced back to the Middle Ages at least. One of its earliest names was chorea,* which, as in "choreography," is the Greek word for dance. The term chorea describes how people affected with the disorder writhe, twist, and turn in a constant, uncontrollable dance-like motion. Later, other descriptive names evolved. "Hereditary chorea" emphasizes how the disease is passed from parent to child. "Chronic progressive chorea" stresses how symptoms of the disease worsen over time. Today, physicians commonly use the simple term Huntington's disease (HD) to describe this highly complex disorder that causes untold suffering for thousands of families.

More than 15,000 Americans have HD. At least 150,000 others have a 50 percent risk of developing the disease and thousands more of their relatives live with the possibility that they, too, might develop HD.

Until recently, scientists understood very little about HD and could only watch as the disease continued to pass from generation to generation. Families saw the disease destroy their loved ones' ability to feel, think, and move. In the last several years, scientists working with support from the National Institute of Neurological Disorders and Stroke (NINDS) have made several breakthroughs in the area of HD research. With these advances, our understanding of the disease continues to improve.

This brochure presents information about HD, and about current research progress, to health professionals, scientists, caregivers, and, most important, to those already too familiar with the disorder: the many families who are affected by HD.

HD results from genetically programmed degeneration of nerve cells, called neurons,* in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance. Specifically affected are cells of the basal ganglia, structures deep within the brain that have many important functions, including coordinating movement. Within the basal ganglia, HD especially targets neurons of the striatum, particularly those in the caudate nuclei and the pallidum. Also affected is the brain's outer surface, or cortex, which controls thought, perception, and memory.

HD is found in every country of the world. It is a familial disease, passed from parent to child through a mutation or misspelling in the normal gene.

A single abnormal gene, the basic biological unit of heredity, produces HD. Genes are composed of deoxyribonucleic acid (DNA), a molecule shaped like a spiral ladder. Each rung of this ladder is composed of two paired chemicals called bases. There are four types of basesadenine, thymine, cytosine, and guanineeach abbreviated by the first letter of its name: A, T, C, and G. Certain bases always "pair" together, and different combinations of base pairs join to form coded messages. A gene is a long string of this DNA in various combinations of A, T, C, and G. These unique combinations determine the gene's function, much like letters join together to form words. Each person has about 30,000 genesa billion base pairs of DNA or bits of information repeated in the nuclei of human cellswhich determine individual characteristics or traits.

Genes are arranged in precise locations along 23 rod-like pairs of chromosomes. One chromosome from each pair comes from an individual's mother, the other from the father. Each half of a chromosome pair is similar to the other, except for one pair, which determines the sex of the individual. This pair has two X chromosomes in females and one X and one Y chromosome in males. The gene that produces HD lies on chromosome 4, one of the 22 non-sex-linked, or "autosomal," pairs of chromosomes, placing men and women at equal risk of acquiring the disease.

The impact of a gene depends partly on whether it is dominant or recessive. If a gene is dominant, then only one of the paired chromosomes is required to produce its called-for effect. If the gene is recessive, both parents must provide chromosomal copies for the trait to be present. HD is called an autosomal dominant disorder because only one copy of the defective gene, inherited from one parent, is necessary to produce the disease.

The genetic defect responsible for HD is a small sequence of DNA on chromosome 4 in which several base pairs are repeated many, many times. The normal gene has three DNA bases, composed of the sequence CAG. In people with HD, the sequence abnormally repeats itself dozens of times. Over timeand with each successive generationthe number of CAG repeats may expand further.

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Huntington's Disease: Hope Through Research: National ...

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IRVINE, CA (PRWEB) December 20, 2013

Proove Biosciences, the leader in providing personalized genetic pain medicine services, participated and presented clinical data and research at the Eastern Pain Associations Annual Meeting on December 7, 2013. The meeting covered current topics in pain medicine and opioid treatment, and took place at the New York Marriott Downtown, in Manhattan.

The EPA offers local and regional scientific meetings to foster an exchange of clinical and scientific information among multidisciplinary health professionals and researchers interested in the field of pain. The Annual EPA Scientific Meeting offered symposia and lectures given by nationally recognized speakers, posters and exhibits designed to appeal to a wide spectrum of specialists and interests.

The EPAs Annual Meeting concluded a successful and busy year for Proove presenting research throughout the country. Most recently, Proove presented data and findings at the ASRAs Pain Medicine Meeting, The National Workers Compensation Conference and Expo, and the Common Sense Pain Management Conference.

Our research and clinical team, along with our aggressive business development efforts have allowed Proove to experience outstanding growth throughout the year, stated Proove Biosciences President and Founder,Brian Meshkin. We are the only company providing proprietary testing services in personalized pain medicine. We are happy to have been a part of the EPAs regional meeting, and exhibiting our work with physicians, psychologists, nurses, and scientists dedicated to pain research.

About Proove Biosciences Proove Biosciences is the leading Personalized Pain Medicine laboratory that provides proprietary genetic testing services to help physicians improve outcomes for patients and contain costs for insurers. With offices in Southern California and the Baltimore-Washington metropolitan area, the Company is the research leader investigating and publishing data on the genetics of pain medicine with clinical research sites across the United States. Physicians use Proove Biosciences testing to improve pain medicine selection, dosing, and evaluation of medications they prescribe. From a simple cheek swab collected in the office, Proove performs proprietary genetic tests in its CLIA-certified laboratory to identify patients at risk for misuse of prescription pain medications and evaluate their metabolism of medications. For more information, please visit http://www.proovebio.com or call toll free 855-PROOVE-BIO (855-776-6832).

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IV International Symposium of Genetics and Breeding - 1/7
Session: Pioneer/GenMelhor partnership PhD Tabare Abadie e Leonardo de Azevedo Peixoto.

By: GenMelhor UFV

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IV International Symposium of Genetics and Breeding - 1/7 - Video

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PhD Student Showcase: Chung Ching Chu, Division of Genetics and Molecular Medicine

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PhD Student Showcase: Chung Ching Chu, Division of Genetics and Molecular Medicine - Video

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Innovation Campaign - Genetics Research

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Dr. Janet Rowley, a pioneer in cancer genetics research, has died at age 88.

Rowley spent most of her career at the University of Chicago, where she also obtained her medical degree. She died Tuesday of ovarian cancer complications at her home nearby, the university said in a statement.

Rowley conducted landmark research with leukemia in the 1970s, linking cancer with genetic abnormalities work that led to targeted drug treatment for leukemia. She identified a genetic process called translocation, now widely accepted. By 1990, more than 70 translocations had been identified in various cancers, according to her biography on the National Library of Medicine's website.

She is a recipient of the National Medal of Science, the nation's highest scientific honor and the Presidential Medal of Freedom, the nation's highest civilian honor.

"Janet Rowley's work established that cancer is a genetic disease," Mary-Claire King, president of the American Society of Human Genetics, said recently. "We are still working from her paradigm."

Rowley, known among colleagues for her intelligence and humility, called receiving the presidential award, in 2009, "quite remarkable."

"I've never regretted being in science and being in research," Rowley said at the time. "The exhilaration that one gets in making new discoveries is beyond description."

With her silvery hair and twinkling eyes, Rowley was a recognizable figure at the University of Chicago, often seen riding her bike around the South Side campus, even up until a few months ago despite her disease. She remained active in research until close to her death and hoped that her own cancer could contribute to understanding of the disease.

Just last month, she was well enough to attend a celebration of the 50th anniversary of the presidential medal in Washington alongside other previous recipients and this year's winners, who include several scientists, former President Bill Clinton, Oprah Winfrey, baseball's Ernie Banks and Loretta Lynn.

Rowley was born in New York City in 1925 and at age 15 won a scholarship to an advanced academic program at the University of Chicago. She went to medical school there when the quota was just three women in a class of 65, the university said.

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Gene therapy for inherited disorders
Gene therapy will be of great significance for patients with hereditary disorders and society at large, says Gerard Wagemaker.

By: youris.com - European Research Media Center

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Rick Taylor, Spinal Cord Injury
When Rick Taylor broke his neck waterskiing, many thought he might never walk again. Dr. Jeffrey Solomon, MD, medical director of inpatient rehabilitation at Asante Rogue Regional, thought...

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Rick Taylor, Spinal Cord Injury - Video

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TMA Podcast Understanding Repair and Recovery After non-traumatic Spinal Cord Injury
This podcast is part of TMA #39;s Ask the Expert podcast series and was held on September 16, 2013 a 1 pm EST. The physician-experts on the panel were Dr. Benjam...

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Los Angeles, Dec 21 : Researchers at UCLA's (University of California, Los Angeles') Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have discovered a mechanism by which certain adult stem cells suppress their ability to initiate skin cancer during their dormant phase an understanding that could be exploited for better cancer-prevention strategies.

The study, which was led by UCLA postdoctoral fellow Andrew White and William Lowry, an associate professor of molecular, cell and developmental biology who holds the Maria Rowena Ross Term Chair in Cell Biology in the UCLA College of Letters and Science, was published online Dec. 15 in the journal Nature Cell Biology.

Hair follicle stem cells, the tissue-specific adult stem cells that generate the hair follicles, are also the cells of origin for cutaneous squamous cell carcinoma, a common skin cancer. These stem cells cycle between periods of activation (during which they can grow) and quiescence (when they remain dormant).

Using mouse models, White and Lowry applied known cancer-causing genes to hair follicle stem cells and found that during their dormant phase, the cells could not be made to initiate skin cancer. Once they were in their active period, however, they began growing cancer.

"We found that this tumor suppression via adult stem cell quiescence was mediated by PTEN, a gene important in regulating the cell's response to signaling pathways," White said.

"Therefore, stem cell quiescence is a novel form of tumor suppression in hair follicle stem cells, and PTEN must be present for the suppression to work."

Understanding cancer suppression through quiescence could better inform preventative strategies for certain patients, such as organ transplant recipients, who are particularly susceptible to squamous cell carcinoma, and for those taking the drug vemurafenib for melanoma, another type of skin cancer.

The study also may reveal parallels between squamous cell carcinoma and other cancers in which stem cells have a quiescent phase.

The research was supported by the California Institute of Regenerative Medicine, the University of California Cancer Research Coordinating Committee and the National Institutes of Health.

--IBNS (Posted on 21-12-2013)

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Adult stem cells suppress cancer while dormant

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(PRWEB) December 21, 2013

Stem cell therapy has a tremendous potential to cure various illnesses and injuries. Recent news items have highlighted possibilities that it could treat damaged spinal cords or revitalize hip joints. Scientists are working on stem cell remedies for dementia, heart disease and diabetes. Doctors in some countries have begun using this therapy to grow replacement body tissue and treat leukemia.

However, stem cell treatments remain controversial. Some people object to them on ethical or religious grounds. Others express concern about the safety of these newfound cures. Animal testing has revealed that minor mistakes can result in impurities that cause cells to produce tumors and other ill effects. Some patients have died after receiving experimental therapies that weren't adequately tested.

The producers of the "Leading Edge" TV series plan to release a new segment that examines this fascinating yet contentious health topic. Presenter Jimmy Johnson will offer an update on important facts and recent developments in the world of stem cell research. Viewers can benefit from the program's concise and unbiased perspective on an issue that many people have yet to learn about.

"Leading Edge" is independently distributed to local public TV broadcasters across the U.S. The national Public Broadcasting Service does not act as its distributor. To learn more about this informational series, please browse http://www.leadingedgeseries.com or send an email message to the program's producers. They can be reached at info(at)leadingedgeseries(dot)com.

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"Leading Edge" Set to Produce New Content Featuring Stem Cell Therapy, with Host Jimmy Johnson

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Florida Hospital Pepin Heart Institute is First in West & Central Florida to Perform a Groundbreaking Stem Cell Clinical Trial for Heart Failure Patients

The first patient has been treated as part of The ATHENA Trial, which derives stem cells from the patientsown adipose (fat) tissue and injects extracted cells into damaged parts of the heart.

TAMPA, Florida (December 20, 2013) Florida Hospital Pepin Heart Institute and Dr. Kiran C. Patel Research Institute announced the first patient, a 59 year old Clearwater man, has been treated as part of the ATHENA clinical trial. The trial, sponsored by San Diego-based Cytori Therapeutics, derives stem cells from the patients own fat tissue and injects extracted cells into damaged parts of the heart. The ATHENA trial is a treatment for chronic heart failure due to coronary heart disease. Dr. Charles Lambert, Medical Director of Florida Hospital Pepin Heart Institute, is leading the way for the first U.S. FDA approved clinical trial using adipose-derived regenerative cells, known as ADRCs, in chronic heart failure patients. I am pleased to report that all procedures went well. The patient is doing well, he was released and is recovering at home. We look forward to following his progress over the coming months, said Dr. Charles Lambert. Heart failure (HF) can occur when the muscles of the heart become weakened and cannot pump blood sufficiently throughout the body. The injury is most often caused by inadequate blood flow to the heart resulting from chronic or acute cardiovascular disease, including heart attacks. The ATHENA clinical trial procedure is a three step process. First, the trial involves the collection of fat from the patients body by liposuction. Then the fat sample is filtered through a machine that extracts out the stem cells. Finally, the stem cells are injected into the damaged part of the patients heart. During this first case at Florida Hospital Pepin Heart Institute, Dr. Paul Smith performed the liposuction to obtain the fat sample, a team at the Dr. Kiran C. Patel Research Institute isolated stem cells from the fat sample and then Dr. Charles Lambert performed the cell therapy by direct injection into the patients heart. Pepin Heart and Dr. Kiran C. Patel Research Institute is exploring and conducting leading-edge research to develop break-through treatments long before they are even available in other facilities. Stem cells have the unique ability to develop into many different cell types, and in many tissues serve as an internal repair system, dividing essentially without limit to replenish other cells, said Dr. Lambert.

The Pepin Heart Institute has a history of cardiovascular stem cell research as part of the NIH sponsored Cardiac Cell Therapy Research Network (CCTRN) as well as other active cell therapy trials. The trial is a double blind, randomized, placebo controlled study designed to study the use of a patients own Adipose-Derived Regenerative Cells (ADRCs) to treat chronic heart failure from coronary heart disease in patients who are on maximal therapy and still have heart failure symptoms. All trial participants undergo a minor liposuction procedure to remove fat (adipose) tissue. Following the liposuction, trial participants may have their tissue processed with Cytoris proprietary Celution System to separate and concentrate cells, and prepare them for therapeutic use. Trial participants will then have either their own cells or a placebo injected back into their damaged heart tissue. To test whether ADRCs will improve heart function, several measurements will be made, including peak oxygen consumption (VO2max), which measures how much physical exercise (gentle walking on a treadmill) a patient can perform, blood flow to the heart (perfusion), the amount of blood in the left ventricle at the end of contraction and relaxation (end-systolic and end-diastolic volumes), and the fraction of blood that is pumped during each contraction (ejection fraction). After the injection procedure, patients are seen in the clinic for follow-up visits over the first 12 months; they are then contacted by phone once a year for up to five years after the procedure.

There are approximately 5.1 million Americans currently living with heart failure, according to the American Heart Association. Chronic heart failure due to coronary heart disease is a severe, debilitating condition caused by restriction of blood flow to the heart muscle, reducing the hearts oxygen supply and limiting its pumping function. Individuals interested in participating in the ATHENA clinical research trial or learning more can visit http://www.theathenatrial.com or call Brian Nordgren, Florida Hospital Pepin Heart Institute Physician Assistant & Stem Cell Program Lead at (813) 615-7527.

About Florida Hospital Tampa Florida Hospital Tampa is a not-for-profit 475-bed tertiary hospital specializing in cardiovascular medicine, neuroscience, orthopaedics, womens services, pediatrics, oncology, endocrinology, bariatrics, wound healing, sleep medicine and general surgery including minimally invasive and robotic-assisted procedures. Also located at Florida Hospital Tampa is the renowned Florida Hospital Pepin Heart Institute, a recognized leader in cardiovascular disease prevention, diagnosis, treatment and leading-edge research. Part of the Adventist Health System, Florida Hospital is a leading health network comprised of 22 hospitals throughout the state. For more information, visit http://www.FHTampa.org.

About Florida Hospital Pepin Heart Institute and Dr. Kiran C. Patel Research Institute Florida Hospital Pepin Heart Institute is a free-standing cardiovascular institute providing comprehensive cardiovascular care with over 76,000 angioplasty procedures and 11,000 open-heart surgeries in the Tampa Bay region. Leading the way with the first accredited chest pain emergency room in Tampa Bay, the institute is among an elite few in the state of Florida chosen to perform the ground breaking Transcatheter Aortic Valve Replacement (TAVR) procedure. It is also a HeartCaring designated provider and a Larry King Cardiac Foundation Hospital. Florida Hospital Pepin Heart Institute and the Dr. Kiran C. Patel Research Institute, affiliated with the University of South Florida (USF), are exploring and conducting leading-edge research to develop break-through treatments long before they are available in most other hospitals. To learn more, visit http://www.FHPepin.org.

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Groundbreaking Stem Cell Clinical Trial

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(PRWEB) December 20, 2013

Florida Hospital Pepin Heart Institute and Dr. Kiran C. Patel Research Institute announced the first patient, a 59 year old Clearwater man, has been treated as part of the ATHENA clinical trial. The trial, sponsored by San Diego-based Cytori Therapeutics, derives stem cells from the patients own fat tissue and injects extracted cells into damaged parts of the heart. The ATHENA trial is a treatment for chronic heart failure due to coronary heart disease. Dr. Charles Lambert, Medical Director of Florida Hospital Pepin Heart Institute, is leading the way for the first U.S. FDA approved clinical trial using adipose-derived regenerative cells, known as ADRCs, in chronic heart failure patients. I am pleased to report that all procedures went well. The patient is doing well, he was released and is recovering at home. We look forward to following his progress over the coming months, said Dr. Charles Lambert.

Heart failure (HF) can occur when the muscles of the heart become weakened and cannot pump blood sufficiently throughout the body. The injury is most often caused by inadequate blood flow to the heart resulting from chronic or acute cardiovascular disease, including heart attacks. The ATHENA clinical trial procedure is a three step process. First, the trial involves the collection of fat from the patients body by liposuction. Then the fat sample is filtered through a machine that extracts out the stem cells. Finally, the stem cells are injected into the damaged part of the patients heart. During this first case at Florida Hospital Pepin Heart Institute, Dr. Paul Smith performed the liposuction to obtain the fat sample, a team at the Dr. Kiran C. Patel Research Institute isolated stem cells from the fat sample and then Dr. Charles Lambert performed the cell therapy by direct injection into the patients heart. Pepin Heart and Dr. Kiran C. Patel Research Institute is exploring and conducting leading-edge research to develop break-through treatments long before they are even available in other facilities. Stem cells have the unique ability to develop into many different cell types, and in many tissues serve as an internal repair system, dividing essentially without limit to replenish other cells, said Dr. Lambert. The Pepin Heart Institute has a history of cardiovascular stem cell research as part of the NIH sponsored Cardiac Cell Therapy Research Network (CCTRN) as well as other active cell therapy trials. The trial is a double blind, randomized, placebo controlled study designed to study the use of a patients own Adipose-Derived Regenerative Cells (ADRCs) to treat chronic heart failure from coronary heart disease in patients who are on maximal therapy and still have heart failure symptoms. All trial participants undergo a minor liposuction procedure to remove fat (adipose) tissue. Following the liposuction, trial participants may have their tissue processed with Cytoris proprietary Celution System to separate and concentrate cells, and prepare them for therapeutic use. Trial participants will then have either their own cells or a placebo injected back into their damaged heart tissue. To test whether ADRCs will improve heart function, several measurements will be made, including peak oxygen consumption (VO2max), which measures how much physical exercise (gentle walking on a treadmill) a patient can perform, blood flow to the heart (perfusion), the amount of blood in the left ventricle at the end of contraction and relaxation (end-systolic and end-diastolic volumes), and the fraction of blood that is pumped during each contraction (ejection fraction). After the injection procedure, patients are seen in the clinic for follow-up visits over the first 12 months; they are then contacted by phone once a year for up to five years after the procedure. There are approximately 5.1 million Americans currently living with heart failure, according to the American Heart Association. Chronic heart failure due to coronary heart disease is a severe, debilitating condition caused by restriction of blood flow to the heart muscle, reducing the hearts oxygen supply and limiting its pumping function. Individuals interested in participating in the ATHENA clinical research trial or learning more can visit http://www.theathenatrial.com or call Brian Nordgren, Florida Hospital Pepin Heart Institute Physician Assistant & Stem Cell Program Lead at (813) 615-7527.

About Florida Hospital Pepin Heart Institute and Dr. Kiran C. Patel Research Institute Florida Hospital Pepin Heart Institute, located at Florida Hospital Tampa, is a free-standing cardiovascular institute providing comprehensive cardiovascular care with over 76,000 angioplasty procedures and 11,000 open-heart surgeries in the Tampa Bay region. Leading the way with the first accredited chest pain emergency room in Tampa Bay, the institute is among an elite few in the state of Florida chosen to perform the ground breaking Transcatheter Aortic Valve Replacement (TAVR) procedure. It is also a HeartCaring designated provider and a Larry King Cardiac Foundation Hospital. Florida Hospital Pepin Heart Institute and the Dr. Kiran C. Patel Research Institute, affiliated with the University of South Florida (USF), are exploring and conducting leading-edge research to develop break-through treatments long before they are available in most other hospitals. To learn more, visit http://www.FHPepin.org

About Cytori Therapeutics Cytori Therapeutics, Inc. is developing cell therapies based on autologous adipose-derived regenerative cells (ADRCs) to treat cardiovascular disease and repair soft tissue defects. Our scientific data suggest ADRCs improve blood flow, moderate the immune response and keep tissue at risk of dying alive. As a result, we believe these cells can be applied across multiple "ischemic" conditions. These therapies are made available to the physician and patient at the point-of-care by Cytori's proprietary technologies and products, including the Celution system product family. http://www.cytori.com

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December 20, 2013

redOrbit Staff & Wire Reports Your Universe Online

Regenerative medicine research conducted throughout this year at the University of Southern California (USC) could lead to new ways to counter baldness and receding hairlines using stem cells.

USC Assistant Professor of Pathology Dr. Krzysztof Kobielak and his colleagues have published a trio of papers in the journals Stem Cells and the Proceedings of the National Academy of Sciences (PNAS) describing some of the biological factors responsible for when hair starts growing, when it stops, and when it falls out.

According to USC, the three studies focused on stem cells that are located in adult hair follicles. Those cells, known as hfSCs, can regenerate both hair follicles and skin, and are governed by bone morphogenetic proteins (BMPs) and the Wnt signaling pathways groups of molecules that work together in order to control the cycles of hair growth and other cellular functions.

The most recent paper, published in the journal Stem Cells in November 2013, focuses on how the gene Wnt7b activates hair growth. Without Wnt7b, hair is much shorter, the team said. Kobielaks team originally proposed Wnt7bs role in a study published this January in PNAS. That paper identified a complex network of genes, including the Wnt and BMP signaling pathways, which controls the cycles of hair growth.

Reduced BMP signaling and increased Wnt signaling activate hair growth, while increased BMP signaling and decreased Wnt signaling keeps the hfSCs in a resting state, the researchers explained. The third paper, published in Stem Cells in September, sheds new light on the BMP signaling pathway. It looked at the function of the proteins Smad1 and Smad 5, which send and receive signals that regulate hair-related stem cells during growth periods.

Collectively, these new discoveries advance basic science and, more importantly, might translate into novel therapeutics for various human diseases, Kobielak explained. Since BMP signaling has a key regulatory role in maintaining the stability of different types of adult stem cell populations, the implication for future therapies might be potentially much broader than baldness and could include skin regeneration for burn patients and skin cancer.

Other USC researchers involved in the studies include postdoctoral fellow Eve Kandyba, Yvonne Leung, Yi-Bu Chen, Randall Widelitz, Cheng-Ming Chuong, Virginia M. Hazen, Agnieszka Kobielak, and Samantha J. Butler. Funding for the research was provided by the Donald E. and Delia B. Baxter Foundation Award and National Institute of Arthritis and Musculoskeletal and Skin Diseases of the National Institutes of Health (NIH).

Source: redOrbit Staff & Wire Reports - Your Universe Online

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Stem Cell Research Could Lead To A Cure For Baldness, And More

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Were in for a thrill, said Chancellor Susan Desmond-Hellman, MD, MPH, as she kicked off a special day-long symposium recognizing the winners of the 2014 Breakthrough Prizes in Life Sciences at Genentech Halls Byers Auditorium on Dec. 13. It was the centerpiece of a two-day celebration hosted by UCSF.

The morning got off to a rousing start with a talk about advancements in cancer genetics. The disease was a mystery when Bert Vogelstein, MD, began his career in the mid-1970s.

Though President Richard Nixon had declared a metaphoric war on cancer, the scientific community didnt know who the enemy was, said Vogelstein, director of the Ludwig Center for Cancer Genetics and Therapeutics at Johns Hopkins School of Medicine.

Chancellor Susan Desmond-Hellmann welcomes attendees

to the daylong symposium.

Forty years later we know our enemy well, he said, having identified about 150 driver genes that exert their effects in about 12 pathways.

In a lecture peppered with military metaphors, Vogelstein said we have moved past the conventional warfare of chemotherapy to targeted therapies, and have recently been recruiting the allies of the immune system. But it is crucial, he said, to begin pre-emptive strikes.

If Plan A of the War on Cancer was finding a way to cure advanced cancers, he said, it is time to initiated Plan B: identifying biomarkers of very early cancers and eliminating them from the body before they take hold and metastasize.

In keeping with Vogelsteins historical approach, Robert Weinberg, PhD, Daniel K. Ludwig Professor for Cancer Research at the Whitehead Institute in Cambridge, Mass., presented a masterful history of cancer biology beginning with the discovery of the first oncogenes in experiments with carcinogenic chemicals.

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Innovative Researchers Honored for Work Fighting Life-Threatening Diseases

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Mayo Clinic to Grow Human Cells in Space to Test Treatment for Stroke
Abba Zubair, M.D., Ph.D, medical and scientific director of the Cell Therapy Laboratory at Mayo Clinic in Florida, talks about the $300000 his research team...

By: Mayo Clinic

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Mayo Clinic to Grow Human Cells in Space to Test Treatment for Stroke - Video

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(PRWEB) December 21, 2013

Stem cell therapy has a tremendous potential to cure various illnesses and injuries. Recent news items have highlighted possibilities that it could treat damaged spinal cords or revitalize hip joints. Scientists are working on stem cell remedies for dementia, heart disease and diabetes. Doctors in some countries have begun using this therapy to grow replacement body tissue and treat leukemia.

However, stem cell treatments remain controversial. Some people object to them on ethical or religious grounds. Others express concern about the safety of these newfound cures. Animal testing has revealed that minor mistakes can result in impurities that cause cells to produce tumors and other ill effects. Some patients have died after receiving experimental therapies that weren't adequately tested.

The producers of the "Leading Edge" TV series plan to release a new segment that examines this fascinating yet contentious health topic. Presenter Jimmy Johnson will offer an update on important facts and recent developments in the world of stem cell research. Viewers can benefit from the program's concise and unbiased perspective on an issue that many people have yet to learn about.

"Leading Edge" is independently distributed to local public TV broadcasters across the U.S. The national Public Broadcasting Service does not act as its distributor. To learn more about this informational series, please browse http://www.leadingedgeseries.com or send an email message to the program's producers. They can be reached at info(at)leadingedgeseries(dot)com.

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"Leading Edge" Set to Produce New Content Featuring Stem Cell Therapy, with Host Jimmy Johnson

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Newswise DALLAS Dec. 19, 2013 UTSouthwestern neuroscience researchers have identified a gene that controls the response to cocaine by comparing closely related strains of mice often used to study addiction and behavior patterns.

The researchers suspect that the newly identified gene, Cyfip2, determines how mammals respond to cocaine, although it is too soon to tell what the indications are for humans or for addiction, said Dr. Joseph Takahashi, chair of neuroscience and a Howard Hughes Medical Institute investigator at UTSouthwestern and the senior author of the study.

The findings, reported in Science, evolved from examining the genetic differences between two substrains of the standard C57BL/6 mouse strain: a J strain from the Jackson Laboratory (C57BL/6J) and an N strain from the National Institutes of Health (C57BL/6N). Researchers compared the two strains of mice and used their differential responses to cocaine to identify the causative gene.

We found that the N strain has accumulated mutations over time, one of which has a very strong effect on cocaine response, Dr. Takahashi said. We propose that CYFIP2 the protein produced by the Cyfip2 gene is a key regulator of cocaine response in mammals.

The Takahashi laboratory has identified about 100 genetic differences that affect protein sequences between the two mouse strains, meaning that there are many genetic differences whose effects are not yet known, he added.

We identified this gene by first using a forward genetics strategy to search for differences in traits between the two mouse strains. We found a difference in cocaine response between them, with the C57BL/6N strain showing a reduced behavioral response, Dr. Takahashi said. We then carried out genetic mapping and whole genome sequencing, which allowed us to pinpoint the Cyfip2 gene as the causative one in a rapid and unambiguous way.

The C57BL/6J J mouse is the gold-standard strain for most research involving the mouse. For example, the reference sequence for the mouse genome, as well as most behavioral and physiological experiments, are based on the J strain. However, the International Knockout Mouse Consortium will be shifting emphasis to the N strain since they have created 17,000 embryonic stem cell lines with gene mutations that originate from the N strain. Thus, identifying genetic differences between these two mouse strains is important, Dr. Takahashi said.

Although mouse geneticists pay close attention to the specific strains of mice that they use, it has not been generally appreciated that sublines of the same strain of mouse might differ so profoundly. Thus, a C57BL/6 mouse might appear to be the same, but in fact there are many, many sublines of this laboratory mouse, and it is important to know which exact one you are using. Since the knockout mouse project has produced so many mutations (17,000) derived from the N strain, it will be even more important to keep in mind that not all C57BL/6 mice are the same.

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UT Southwestern Neuroscience Researchers Identify Gene Involved in Response to Cocaine

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Are GMOs Safe Medical Course
For Educational Use Only - Fair Use - E.R. physician Dr. Travis Stork explains how GMOs Genetically Modified Organisms amplify crop production and sustainabi...

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An American activist opposing genetic engineering has praised Nelson as the first part of New Zealand to declare itself free of genetically modified organisms.

Self-published author and speaker Jeffrey Smith gave a talk at the Free House pub this week emphasising the value in keeping genetically engineered products out of New Zealand. It was one of only two talks he gave nationwide.

"New Zealand is very well-poised to take advantage of the economics of going non-GM."

He said there was a growing sentiment in his homeland that genetically modified products should be avoided. He expected a consumer-driven "tipping point" to occur within the next 18 months, saying this would see products containing GM ingredients becoming a "commercial liability".

"At that point, the clean, green image of New Zealand will translate better into economic premiums."

Mr Smith said there was a particularly receptive market available for meat and dairy products which originated from animals that had not eaten GM feed. New Zealand farmers should phase out the use of GM feed and market their meat and dairy in the US, claiming the GE free products would command a premium.

In New Zealand, processed foods can contain GM ingredients but must be labelled accordingly. No GM crops are grown commercially and no GM fruit, vegetables or meat are sold, but meat and other products from animals that have been fed GM food are not required to be labelled.

Mr Smith claimed GE foods had been found to cause health problems, but said studies into this area had been suppressed.

He was not all praise for New Zealand, criticising the local processes in place for the approval of GE products. He said the process was "nowhere near" rigorous enough and did not protect the public, saying it was widely cited internationally as an example of "how regulations should not be conducted".

Based in Iowa, Mr Smith was hosted in New Zealand by non-profit organisation GE Free New Zealand. President Claire Bleakley said it would be enlightening for a local audience to gain insights on the international experience with genetic modification.

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Activist lauds GE-free city

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Most popular Genetic Medicine Austin TX
Hiring authorities actually mean alot as a result of that, we ensure our Genetic Medicine solutions are completed with professionals and specialist strategies. http://www.localpros.us/austin-tx/gen...

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PUBLIC RELEASE DATE:

20-Dec-2013

Contact: Jessica Studeny jessica.studeny@case.edu 216-368-4692 Case Western Reserve University

Many rare disorders are caused by gene mutation, like sickle cell anemia. Yet until now the underlying genetic cause of more common conditions for example, rheumatoid arthritis has evaded scientists for years.

New research from Case Western Reserve University School of Medicine to appear in the journal Genome Research finds that six common diseases arise from DNA changes located outside genes. The study from the laboratory of Peter Scacheri, PhD, shows that multiple DNA changes, or variants, work in concert to affect genes, leading to autoimmune diseases including rheumatoid arthritis, Crohn's disease, celiac disease, multiple sclerosis, lupus and colitis. Further, for each disease, multiple different genes are manipulated by several small differences in DNA.

"We've known that rare diseases are due to one change within one gene with major effects. The key take away is that common diseases are due to many changes with small effects on a handful of genes," said Scacheri, associate professor of genetics and genome sciences.

The research is in advanced online publication and can be found at http://tinyurl.com/okml3ag.

The human genome includes 3 billion letters of DNA. Only 1 to 2 percent of the letters are used as the blueprint for proteins, the body's building blocks. Scacheri's team is part of group of scientists investigating where and why DNA goes awry in the remaining 98 percent the regions between genes. These regions contain thousands of genetic switches that control the levels of genes. This new finding shows that in common diseases, the fine-tuning of those switches is not quite right, leading to incorrect expression of some key genes previously unidentified.

"This is a paradigm shift for the field with respect to pinpointing the genetic causes of common disease susceptibility," Scacheri said.

"The Scacheri lab's study provides a new model for understanding how genetic variants explain variation in common, complex diseases such as rheumatoid arthritis and colitis. That is, the effect of an individual variant may be very small, but when coupled with other nearby variants, the manifestations are much greater, said Anthony Wynshaw-Boris, MD, PhD, chair of the Department of Genetics and Genome Sciences at Case Western Reserve University School of Medicine and University Hospitals Case Medical Center and the James H. Jewell MD '34 Professor of Genetics at the School of Medicine. "This model may also help to explain why genetic studies of these and other common diseases have so far fallen short of providing a satisfactory explanation of the genetic pathways important for the development of these disorders."

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Common disorders: It's not the genes themselves, but how they are controlled

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London, UK (PRWEB) December 20, 2013

Nowadays, molecular diagnostics in generic testing introduces advanced analytical techniques to the treatment and diagnosis of various generic disorders. The burgeoning growth in the market is propelled by considerable breakthroughs in proteomics and genomics, as well as by the ongoing development of microarray devices to measure different analytes within body tissues and blood. The range of the major recent developments includes but is not limited to the introduction and rapid uptake of cell-free fetal DNA prenatal testing, advancements in the development of personalised medicine, the integration of gene expression profiling and specialty labs into clinical practice, expansion of the installed base of automated instruments used for molecular testing and also the considerable progress of companion diagnostics for drug development.

At present, generic testing forms one of the most profitable segments of the overall molecular diagnostics space. The generic testing area has a huge growth potential, which is poised to be the major area of interest in the upcoming years.

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The in-demand report presents a comprehensive guide to the emerging market for molecular diagnostics in generic testing globally and in the USA. It provides an in-depth assessment of this emerging field, offers a detailed analysis of its performance, estimates the size and growth potential of the molecular diagnostics in the genetic testing area. The research study contains a detailed examination of the major factors influencing the development of different market sectors, delves into the competitive environment and uncovers vital information on the performance of the leading companies engaged in the industry. The report characterises the regulative framework, describes the current market landscape and then thoroughly discusses the future growth prospects of molecular diagnostics in the generic testing universe.

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Molecular Diagnostics Prospects in Genetic Testing Area Discussed in Discounted Report by TriMark Published at ...

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Bicuspid aortic valve: Prevalence, genetics and natural history.

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