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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.
Yale researchers successfully correct gene mutation that causes cystic fibrosis

Yale researchers successfully correct gene mutation that causes cystic fibrosis

Yale researchers successfully corrected the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder. The study was published April 27 in Nature Communications. [More]
Gene therapy reduces infectious complications in children, teens with Wiskott-Aldrich syndrome

Gene therapy reduces infectious complications in children, teens with Wiskott-Aldrich syndrome

In a small study that included seven children and teens with Wiskott-Aldrich syndrome, a rare immunodeficiency disorder, use of gene therapy resulted in clinical improvement in infectious complications, severe eczema, and symptoms of autoimmunity, according to a study in the April 21 issue of JAMA, a theme issue on child health. [More]
UCLA-led stroke study selected as one of 10 most outstanding research papers by CRF

UCLA-led stroke study selected as one of 10 most outstanding research papers by CRF

A UCLA-led study on improving stroke care was selected by the Clinical Research Forum (CRF) as one of the 10 most outstanding research papers written by teams from across the nation in 2014. The organization highlighted the papers at its fourth annual awards ceremony in Washington, D.C. on April 16. [More]
Global changes in cancer cells' epigenome may determine disease progression

Global changes in cancer cells' epigenome may determine disease progression

Genomic studies have illuminated the ways in which malfunctioning genes can drive cancer growth while stunting the therapeutic effects of chemotherapy and other treatments. But new findings from Weill Cornell Medical College investigators indicate that these genes are only partly to blame for why treatment that was at one point effective ultimately fails for about 40 percent of patients diagnosed with the most common form of non-Hodgkin Lymphoma. [More]
New Dream Team dedicated to ovarian cancer research formed at AACR Annual Meeting 2015

New Dream Team dedicated to ovarian cancer research formed at AACR Annual Meeting 2015

Stand Up To Cancer, Ovarian Cancer Research Fund, Ovarian Cancer National Alliance, and National Ovarian Cancer Coalition, along with the American Association for Cancer Research, Scientific Partner to SU2C, announced today the formation of a Dream Team devoted to ovarian cancer research at the AACR Annual Meeting 2015, held here April 18-22. [More]
University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) awarded University of Washington a $148,000 grant to continue the functional analysis of spectrin-like repeats in dystrophin. [More]
RetroSense Therapeutics receives Luis Villalobos Award

RetroSense Therapeutics receives Luis Villalobos Award

RetroSense Therapeutics, LLC, a Wayne State University start-up biotechnology company, has received the Luis Villalobos Award from the Angel Capital Association, the world's leading professional association for angel investors. [More]
Donald S. Coffey to receive Margaret Foti Award at AACR Annual Meeting 2015

Donald S. Coffey to receive Margaret Foti Award at AACR Annual Meeting 2015

Donald S. Coffey, PhD, will be honored with the ninth annual American Association for Cancer Research Margaret Foti Award for Leadership and Extraordinary Achievements in Cancer Research at the AACR Annual Meeting 2015, to be held in Philadelphia, April 18-22. [More]
Autophagy: A new approach to fighting tuberculosis

Autophagy: A new approach to fighting tuberculosis

A new approach to combatting tuberculosis would take advantage of a complex, natural process called autophagy that the human body uses to recycle nutrients, remove damaged cell components, eliminate invading bacteria, and respond to inflammation. [More]
Penn researchers receive 2015 Vaccine Industry Excellence Award

Penn researchers receive 2015 Vaccine Industry Excellence Award

The laboratory of David Weiner, PhD, a professor of Pathology and Laboratory Medicine at the Perelman School of Medicine at the University of Pennsylvania, received the 2015 Vaccine Industry Excellence Award for Best Academic Research Team, at the World Vaccine Congress in Washington, DC this week. [More]
UAB scientists create alternative to the use of viruses in gene therapy

UAB scientists create alternative to the use of viruses in gene therapy

A team of scientists from the Institute for Biotechnology and Biomedicine at the UAB has produced an alternative to the use of viruses in gene therapy. The researchers synthesised nanoparticles which act as artificial viruses, capable of surrounding DNA fragments and releasing them as therapeutic agents, with no biological risk, into the interior of the cells. [More]
Experimental oligonucleotide-based drug stimulates human immune system to fight infections

Experimental oligonucleotide-based drug stimulates human immune system to fight infections

An experimental single-stranded oligonucleotide-based drug, MGN1703, comprised only of natural DNA components, stimulates the human immune system to fight infections and attack cancer cells without causing the harmful side effects associated with similar compounds that also contain non-natural DNA components. [More]
Society for Reproductive Investigation awards two top honors to Dr. Ayman Al-Hendy

Society for Reproductive Investigation awards two top honors to Dr. Ayman Al-Hendy

Dr. Ayman Al-Hendy, an obstetrician-gynecologist and molecular biologist at the Medical College of Georgia at Georgia Regents University and GRHealth, has received two top honors from the Society for Reproductive Investigation. [More]
MDC scientists find solution to increase efficiency of precise genetic modifications

MDC scientists find solution to increase efficiency of precise genetic modifications

CRISPR-Cas9 is a powerful new tool for editing the genome. For researchers around the world, the CRISPR-Cas9 technique is an exciting innovation because it is faster and cheaper than previous methods. Now, using a molecular trick, Dr. Van Trung Chu and Professor Klaus Rajewsky of the Max Delbrück Center for Molecular Medicine Berlin-Buch and Dr. Ralf Kühn, MDC and Berlin Institute of Health, have found a solution to considerably increase the efficiency of precise genetic modifications by up to eightfold. [More]
Novasep, Celladon sign agreement to develop and supply MYDICAR drug substance

Novasep, Celladon sign agreement to develop and supply MYDICAR drug substance

Novasep, a leading supplier of services and technologies for the life sciences and chemical industries, and Celladon Corporation, a clinical-stage cardiovascular gene therapy company, today announce that they have signed a Development, Manufacturing and Supply Agreement pursuant to which, if supported by upcoming MYDICAR clinical data, Novasep would manufacture MYDICAR drug substance through 2018 with extension options through 2020. [More]
Personalized gene therapy to fight against cancer

Personalized gene therapy to fight against cancer

The fight to treat cancer and eradicate tumors will likely benefit from a new set of treatments if early development phases continue to show promise, according to Kalorama Information. The healthcare market research publisher stated that gene therapies that are able to deliver genetic material to a specific cell population or tumor that will result in the destruction of the tumor. [More]
Experimental drug that attacks brain tumor cells passes early tests

Experimental drug that attacks brain tumor cells passes early tests

An experimental drug that attacks brain tumor tissue by crippling the cells' energy source called the mitochondria has passed early tests in animal models and human tissue cultures, say Houston Methodist scientists. [More]
Stempeutics' Stempeucel drug receives ATMP classification from EMA for treatment of TAO

Stempeutics' Stempeucel drug receives ATMP classification from EMA for treatment of TAO

Stempeutics Research, a group company of Manipal Education and Medical Group and a Joint Venture with Cipla Group, announced today that the European Medicines Agency has granted Advanced Therapy Medicinal Product classification for its novel stem cell drug 'Stempeucel' which will be used for the treatment of Thromboangiitis Obliterans (TAO). [More]
Researchers find key step in understanding genetic mechanism of plants' environmental adaptability

Researchers find key step in understanding genetic mechanism of plants' environmental adaptability

A fundamental question pursued by plant scientists worldwide for the past decade has been answered by researchers led by the University of Sydney in Australia. [More]
Researchers identify new gene variants that could contribute to sporadic motor neurone disease

Researchers identify new gene variants that could contribute to sporadic motor neurone disease

Researchers have identified a new host of gene variants that could make people vulnerable to sporadic motor neurone disease, according to a report published today in the journal, Scientific Reports. [More]
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