Biology News Net - Gene Therapy

New American Chemical Society podcast: Genetically-engineered spider silk for gene therapy

Wed, 16 Nov 2011 19:19:01 -0500

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.

Gene therapy shows promise as hemophilia treatment in animal studies

Thu, 03 Nov 2011 18:11:58 -0500

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.

NIH-funded researchers correct sickle cell disease in adult mice

Thu, 13 Oct 2011 17:21:38 -0500

National Institutes of Health-funded scientists have corrected sickle cell disease in adult laboratory mice by activating production of a special blood component normally produced before, but not after, birth.

First successful double-blind trial of gene therapy for advanced Parkinson's

Fri, 18 Mar 2011 10:49:44 -0500

A multi-center gene therapy trial for patients with advanced Parkinson’s disease demonstrated reduced symptoms of the progressive movement disorder, according to a new study published in Lancet Neurology. The study was designed to deliver the gene for glumatic acid decarboxylase (GAD) packaged in inert viral vectors into an area of the brain called the subthalamic nucleus. GAD makes an important inhibitory chemical called GABA. The subthalamic nucleus is abnormally activated in Parkinson’s disease and this activity leads to the debilitating movement problems. The idea of the gene therapy is that the billions of AAV-2 GAD viral vectors delivered into the subthalamic nucleus will increase GABA, thereby quieting this brain region.

Nanodisk gene therapy

Wed, 12 Jan 2011 01:23:27 -0500

One of the challenges of gene therapy - a set of methodologies aimed at treating several nucleic acid diseases (DNA or RNA) - is to assure that this material arrives directly to the nucleus of the cell without losing a substantial amount along the way and without producing any undesired side effects. With this aim, scientists experiment with the use of different types of vectors, molecules capable of transporting genetic material to the correct place. Presently, natural "deactivated" viruses are the most commonly used vectors in clinical trials, their side effects however often limit therapeutic application.

Targeted therapy reactivates 'guardian of the genome' in resistant cancer

Mon, 15 Nov 2010 22:28:53 -0500

It is common for cancer cells to find some way to disarm p53, also known as "guardian of the genome" due to its action in preventing defective cells from dividing. "The critical importance of the protective function of p53 is underscored by the diversity of molecular strategies employed by cancer cells to subvert p53 activity, such as overexpression of antagonistic proteins like HDM2 and HDMX," explains senior study author Dr. Loren D. Walensky from Harvard Medical School. "Restoration of p53 activity remains an important goal in the quest for more effective cancer therapeutics."

Gene therapy reveals unexpected immunity to dystrophin in patients with Duchenne muscular dystrophy

Thu, 07 Oct 2010 04:01:19 -0500

An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.

Breakthrough gene therapy prevents retinal degeneration

Tue, 17 Aug 2010 01:06:24 -0500

In one of only two studies of its kind, a study from researchers at Tufts University School of Medicine and the Sackler School of Graduate Biomedical Sciences at Tufts demonstrates that non-viral gene therapy can delay the onset of some forms of eye disease and preserve vision. The team developed nanoparticles to deliver therapeutic genes to the retina and found that treated mice temporarily retained more eyesight than controls. The study, published online in advance of print in Molecular Therapy, brings researchers closer to a non-viral gene therapy treatment for degenerative eye disorders.

Gene therapy breakthrough heralds treatment for beta-thalassemia

Tue, 13 Jul 2010 21:38:44 -0500

Italian scientists pioneering a new gene transfer treatment for the blood disorder β-thalassemia have successfully completed preclinical trials, claiming they can correct the lack of beta-globin (ß-globin) in patients' blood cells which causes the disease. The research, published in EMBO Molecular Medicine, reveals how gene therapy may represent a safe alternative to current cures that are limited to a minority of patients.

Scientists design new delivery device for gene therapy

Tue, 06 Jul 2010 19:14:19 -0500

Scientists have designed a nanoparticle that appears to effectively deliver genetic material into cells with minimal toxic effects.