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.

Cases of two AIDS patients renew hope of AIDS cure for many scientists, New York Times reports

The New York Times profiles two AIDS patients whose cases "suggest to many scientists that curing AIDS may be achievable," according to the newspaper. "One man, the so-called Berlin patient, apparently has cleared his HIV infection, albeit by arduous bone marrow transplants," and the other, "a 50-year-old man in Trenton, N.J., underwent a far less difficult gene therapy procedure. While he was not cured, his body was able to briefly control the virus after he stopped taking the usual antiviral drugs, something that is highly unusual," the newspaper writes.

29 Nov 2011

Scientists use genetic methods to successfully repair cleft lips in mice embryos

Scientists at Weill Cornell Medical College used genetic methods to successfully repair cleft lips in mice embryos specially engineered for the study of cleft lip and cleft palate. The research breakthrough may show the way to prevent or treat the conditions in humans.

29 Nov 2011

Study reveals specific functional deficit in inner ear mechanotransduction channels

National Institutes of Health-funded researchers have identified two proteins that may be the key components of the long-sought after mechanotransduction channel in the inner ear-the place where the mechanical stimulation of sound waves is transformed into electrical signals that the brain recognizes as sound.

22 Nov 2011

Gene-therapy trial to restore hearing in deaf mice

Researchers have found long-sought genes in the sensory hair cells of the inner ear that, when mutated, prevent sound waves from being converted to electric signals - a fundamental first step in hearing. The team, co-led by Jeffrey Holt, PhD, in the department of otolaryngology at Children-s Hospital Boston, and Andrew Griffith, MD, PhD, of the NIH-s National Institute on Deafness and other Communication Disorders (NIDCD), then restored these electrical signals in the sensory cells of deaf mice by introducing normal genes.

22 Nov 2011

Older home health care patients are prescribed PIMs three times higher than others

Older adults receiving home health care may be taking a drug that is unsafe or ineffective for someone their age. In fact, nearly 40 percent of seniors receiving medical care from a home health agency are taking at least one prescription medication that is considered potentially inappropriate to seniors, a new study in the Journal of General Internal Medicine has revealed.

22 Nov 2011

Modified spider silk proteins for cancer gene therapy

A new episode in the American Chemical Society's (ACS) award-winning "Global Challenges/Chemistry Solutions" podcast series spins a real-life tale in which spider silk shows promise for overcoming a major barrier to the use of gene therapy in everyday medicine.

17 Nov 2011

Aurora kinase inhibitor may treat neuroendocrine prostate cancer

An international team of researchers led by clinicians at Weill Cornell Medical College have discovered a genetic Achilles' heel in an aggressive type of prostate cancer -- a vulnerability they say can be attacked by a targeted drug that is already in clinical trials to treat other types of cancers.

17 Nov 2011

CF Foundation, Genzyme announce new drug research agreement

Genzyme, a Sanofi company, and Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, today announced a research agreement to support the discovery of new drugs to treat people with the most common mutation found in patients with CF, Delta F508.

17 Nov 2011

EMA grants orphan designation to AMT for hemophilia B gene therapy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMA) has granted orphan designation for its gene therapy program for the treatment of hemophilia B. Orphan designation in the European Union provides several benefits including 10 years of market exclusivity from product launch and access to the central authorization procedure.

16 Nov 2011

EPO-secreting engineered vessels could reverse anemia

Patients who rely on recombinant, protein-based drugs must often endure frequent injections, often several times a week, or intravenous therapy. Researchers at Children's Hospital Boston demonstrate the possibility that blood vessels, made from genetically engineered cells, could secrete the drug on demand directly into the bloodstream.

15 Nov 2011

Lentigen, UH Case Medical Center initiate LG631 Phase I trial in glioblastoma

University Hospitals (UH) Case Medical Center, Case Western Reserve University School of Medicine and Lentigen Corporation announced today the initiation of a novel Phase I clinical trial of LG631 gene therapy for the protection of hematopoietic stem cells (HSCs) from the dose limiting toxicity of chemotherapy with Temodar.

15 Nov 2011

Scientists discover promising approaches for treatment of pain

Scientists are discovering promising approaches to treating pain, one of the most common and debilitating neurological complaints, according to research released today at Neuroscience 2011, the annual meeting of the Society for Neuroscience and the world's largest source of emerging news about brain science and health.

15 Nov 2011

Damaged mitochondria equivalent to an environmental disaster in the cell

Current thinking about Parkinson's disease is that it's a disorder of mitochondria, the energy-producing organelles inside cells, causing neurons in the brain's substantia nigra to die or become impaired.

11 Nov 2011

Clinical diagnoses of autism may vary widely across centers

To diagnose autism spectrum disorders, clinicians typically administer a variety of tests or scales and use information from observations and parent interviews to classify individuals into subcategories listed in standard psychiatric diagnostic manuals.

10 Nov 2011

New genetic approach to treat fibrodysplasia ossificans progressiva

Scientists at Penn's Perelman School of Medicine Center for Research in FOP and Related Disorders have developed a new genetic approach to specifically block the damaged copy of the gene for a rare bone disease, while leaving the normal copy untouched.

9 Nov 2011

Gene therapy and stem cell transplantation successfully reverse hemophilia A

For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients.

3 Nov 2011

Clinical-grade viral vector can cure choroideremia

Researchers at Ohio State University Medical Center and Nationwide Children's Hospital have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males.

3 Nov 2011

Fierce Vaccines highlights Inovio's VGX-3100 as one of 10 Promising Therapeutic Vaccines

Inovio Pharmaceuticals, Inc., which is advancing synthetic vaccines to fight cancers and infectious diseases, announced today that VGX-3100, its investigational SynCon vaccine for treating cervical dysplasia and cancer caused by human papillomavirus, was highlighted by Fierce Vaccines as one of 10 Promising Therapeutic Vaccines.

31 Oct 2011

Mayo Clinic, Oxford BioMedica collaborate to develop gene therapy for glaucoma

Oxford BioMedica plc, the leading gene-based biopharmaceutical company, today announces that it has entered into a research and development collaboration with Mayo Clinic, Rochester (USA) to develop a novel gene therapy for the treatment of chronic glaucoma.

31 Oct 2011

Researchers uncover biochemical signals that trigger generation of new lung alveoli

Researchers at Weill Cornell Medical College say they have taken an important step forward in their quest to "turn on" lung regeneration -- an advance that could effectively treat millions of people suffering from respiratory disorders.

29 Oct 2011