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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
TxCell to highlight success factors in cell therapy research at Cell & Gene Therapy Forum 2015

TxCell to highlight success factors in cell therapy research at Cell & Gene Therapy Forum 2015

TxCell SA, a biotechnology company developing innovative, cost-effective, personalized T cell immunotherapies using antigen specific regulatory T-cells (Ag-Tregs) for severe chronic inflammatory and autoimmune diseases, announces today that Dr. Miguel Forte, Sr. VP Clinical Development and Regulatory Affairs, TxCell, will present at the Cell & Gene Therapy Forum 2015, January 26 - 28, 2015 at Grand Hayatt in Washington DC, United States. [More]
New Baylor research explores novel non-invasive technology for pediatric eye exams

New Baylor research explores novel non-invasive technology for pediatric eye exams

Picture a toddler getting his first eye exam. He's seated in a strange room, with strange instruments and strange bright lights. He can't sit still or open his eyes long enough for that diagnostic poof of air - especially if he has trouble seeing anyway, as children with achromatopsia do. [More]
Umbilical cord-derived stem cells from women with gestational diabetes show premature aging

Umbilical cord-derived stem cells from women with gestational diabetes show premature aging

Multipotent cells isolated from the human umbilical cord, called mesenchymal stromal cells (hUC-MSCs) have shown promise for use in cell therapy to treat a variety of human diseases. However, intriguing new evidence shows that hUC-MSCs isolated from women with gestational diabetes demonstrate premature aging, poorer cell growth, and altered metabolic function, as reported in an article in Stem Cells and Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. [More]
Penn, Spark Therapeutics expand strategic relationship to develop treatment for progressive blindness

Penn, Spark Therapeutics expand strategic relationship to develop treatment for progressive blindness

The University of Pennsylvania has announced an expanded relationship with Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating, genetic diseases. [More]
Researchers reveal key factor in understanding elevated cancer risk linked to gene therapy

Researchers reveal key factor in understanding elevated cancer risk linked to gene therapy

National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome Research Institute, part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation. [More]
Researchers uncover new gene tied to incurable eye disorder

Researchers uncover new gene tied to incurable eye disorder

An exhaustive hereditary analysis of a large Louisiana family with vision issues has uncovered a new gene tied to an incurable eye disorder called retinitis pigmentosa, according to an examination led by scientists at The University of Texas Health Science Center at Houston. [More]
Scientists find that blocking hormone reduces stress-induced infertility in female rats

Scientists find that blocking hormone reduces stress-induced infertility in female rats

University of California, Berkeley, scientists have discovered that chronic stress activates a hormone that reduces fertility long after the stress has ended, and that blocking this hormone returns female reproductive behavior to normal. [More]
New UCLA study sheds light on why some people develop PTSD

New UCLA study sheds light on why some people develop PTSD

Why do some people develop post-traumatic stress disorder (PTSD) while others who suffered the same ordeal do not? A new UCLA discovery may shed light on the answer. [More]
New findings may lay groundwork of novel treatment for people with Huntington's disease

New findings may lay groundwork of novel treatment for people with Huntington's disease

By adjusting the levels of a key signaling protein, researchers improved motor function and brain abnormalities in experimental animals with a form of Huntington's disease, a severe neurodegenerative disorder. [More]
Spark Therapeutics files Form S-1 registration statement for initial public offering of common stock

Spark Therapeutics files Form S-1 registration statement for initial public offering of common stock

Spark Therapeutics announced today that it has filed a registration statement on Form S-1 with the U.S. Securities and Exchange Commission relating to the proposed initial public offering of its common stock. [More]
New therapeutic approach shows potential for treating cancer

New therapeutic approach shows potential for treating cancer

Case Western Reserve researchers have identified a two-pronged therapeutic approach that shows great potential for weakening and then defeating cancer cells. [More]
Researchers treat myocardial infarction with new telomerase-based gene therapy

Researchers treat myocardial infarction with new telomerase-based gene therapy

The enzyme telomerase repairs cell damage produced by ageing, and has been used successfully in therapies to lengthen the life of mice. Now it has been observed that it could also be used to cure illnesses related to the ageing process. [More]
High-dose flu vaccine better than regular flu shot for frail, older adults of long-term care facilities

High-dose flu vaccine better than regular flu shot for frail, older adults of long-term care facilities

The high-dose flu vaccine is significantly better than the regular flu shot at boosting the immune response to the flu virus in frail, older residents of long-term care facilities, according to the results of a University of Pittsburgh School of Medicine study. [More]
Scientists demonstrate technique for editing genome in sperm-producing adult stem cells

Scientists demonstrate technique for editing genome in sperm-producing adult stem cells

Scientists at Indiana University and colleagues at Stanford and the University of Texas have demonstrated a technique for "editing" the genome in sperm-producing adult stem cells, a result with powerful potential for basic research and for gene therapy. [More]
Study finds that K13 gene mutations cause malaria drug resistance in Southeast Asia

Study finds that K13 gene mutations cause malaria drug resistance in Southeast Asia

Growing resistance to malaria drugs in Southeast Asia is caused by a single mutated gene inside the disease-causing Plasmodium falciparum parasite, according to a study led by David Fidock, PhD, professor of microbiology & immunology and of medical sciences (in medicine) at Columbia University Medical Center. [More]
CRISPR/Cas9 genome editing: an interview with Maja Petkovic

CRISPR/Cas9 genome editing: an interview with Maja Petkovic

RNA guided CRISPR nucleases have a great potential for genome modification in many different organisms. [More]
Spark Therapeutics, Pfizer partner to develop SPK-FIX for potential treatment of hemophilia B

Spark Therapeutics, Pfizer partner to develop SPK-FIX for potential treatment of hemophilia B

Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today that it has entered into a global collaboration with Pfizer Inc. for the development and potential commercialization of SPK-FIX, a development program advancing proprietary, bio-engineered adeno-associated virus (AAV) vectors for the potential treatment of hemophilia B. [More]
Henry Ford Hospital recruits patients to participate in brain cancer clinical trial

Henry Ford Hospital recruits patients to participate in brain cancer clinical trial

Henry Ford Hospital today announced that it is actively recruiting patients to participate in a clinical trial taking place at the Hermelin Brain Tumor Center which is showing promising results in patients with brain cancer. [More]

New study explores reliability of optogenetics as method of intervention of temporal lobe seizures

Optogenetics is one of the hottest tools in biomedical research today, a method that uses gene therapy to deliver light-sensitive proteins into specific cells. This new tool allows researchers to interact with a single cell or a network of cells with exquisite precision. Whereas imaging and other technologies allow researchers to watch the brain in action, optogenetics enables them to influence those actions. [More]
Genetics may play major role in Lou Gehrig's disease, study reveals

Genetics may play major role in Lou Gehrig's disease, study reveals

Genetics may play a larger role in causing Lou Gehrig's disease than previously believed, potentially accounting for more than one-third of all cases, according to one of the most comprehensive genetic studies to date of patients who suffer from the condition also known as amyotrophic lateral sclerosis, or ALS. [More]