Gene Therapy News and Research

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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.

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.

21 Apr 2015

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.

20 Apr 2015

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.

20 Apr 2015

Scientists discover protein that boosts immunity to viruses and cancer

Experiments in mice and human cells have shown that the protein promotes the proliferation of cytotoxic T cells, which kill cancer cells and cells infected with viruses...

17 Apr 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.

17 Apr 2015

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.

16 Apr 2015

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.

12 Apr 2015

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.

9 Apr 2015

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.

8 Apr 2015

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.

30 Mar 2015

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.

30 Mar 2015

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.

26 Mar 2015

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.

25 Mar 2015

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.

25 Mar 2015

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).

23 Mar 2015

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.

23 Mar 2015

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.

17 Mar 2015

'Turbocharged' protein holds promise in hemophilia gene therapy

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response.

13 Mar 2015

Study: New gene therapy safe, effective for patients with hemophilia B

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding.

12 Mar 2015

Finding could lead to more effective, less invasive treatment for 'bubble boy' disease

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.