Gene therapy to replace the faulty CLN2 gene, which causes a neurodegenerative disease that is fatal by age 8-12 years, was able to slow significantly the rate of neurologic decline in treated children, according to a paper published online ahead of print in the May 2008 issue (Vol. 19 No. 5) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc.

Promising results from a team of NewYork-Presbyterian Hospital/Weill Cornell Medical Center physician-scientists show that gene therapy is both safe and effective at slowing the progression of Batten disease, or Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL), a rare, genetic, degenerative neurological disorder that usually becomes fatal in children by the age of 8 to 12.

Using gene therapy, plastic surgeons have delivered cancer fighting proteins through skin flaps placed on cancerous tumors on rats with a 79 percent reduction in tumor volume, according to a study in the May issue of Plastic and Reconstructive Surgery®, the official medical journal of the American Society of Plastic Surgeons (ASPS).

In a clinical trial at The Children’s Hospital of Philadelphia, researchers from The University of Pennsylvania have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness. Although the patients have not achieved normal eyesight, the preliminary results set the stage for further studies of an innovative treatment for this and possibly other retinal diseases.

A revolutionary cancer treatment using microscopic magnets to enable 'armed' human cells to target tumours has been developed by researchers funded by the Biotechnology and Biological Sciences Research Council (BBSRC). Research published online in the journal, Gene Therapy, shows that inserting these nano-magnets into cells carrying genes to fight tumours, results in many more cells successfully reaching and invading malignant tumours.

A revolutionary cancer treatment using microscopic magnets to enable 'armed' human cells to target tumours has been developed by researchers funded by the Biotechnology and Biological Sciences Research Council (BBSRC). Research published online today (17 April) in the journal, Gene Therapy, shows that inserting these nanomagnets into cells carrying genes to fight tumours, results in many more cells successfully reaching and invading malignant tumours.

Researchers at the U.S. Department of Energy's Brookhaven National Laboratory have shown that increasing the brain level of receptors for dopamine, a pleasure-related chemical, can reduce use of cocaine by 75 percent in rats trained to self-administer it.

Gene therapy, in which a working gene is inserted into a cell to replace a faulty or absent gene, is a promising experimental technique for the prevention and treatment of disease.

Investigators at Hospital for Special Surgery have identified a new mechanism involved in the pathogenesis of inflammatory diseases such as rheumatoid arthritis. The mechanism may also shed some light on why gene therapy experiments that use adenoviruses to deliver genes to humans have run into problems. The study will appear online on March 16 in the journal Nature Immunology.

Investigators in The Research Institute at Nationwide Children’s Hospital have identified the role of a protein that could potentially lead to new clinical treatments to combat musculoskeletal diseases, including Duchenne muscular dystrophy (DMD).

Researchers from the University of Glasgow have solved a scientific riddle that could lead to the development of more effective gene therapies.

Henry Ford Hospital is embarking on an expanded major clinical trial involving the use of gene therapy in combination with radiation therapy, to determine if the combined treatment is more effective than radiation therapy alone for patients with intermediate risk prostate cancer.