Early stage gene therapy clinical trials are recruiting patients from the developing world, providing medically deprived populations access to interventions that show promise but have largely unknown effects in humans.

Chemists in Japan report development of the world's first DNA molecule made almost entirely of artificial parts. The finding could lead to improvements in gene therapy, futuristic nano-sized computers, and other high-tech advances, they say. Their study is scheduled for the July 23 issue of the Journal of the American Chemical Society, a weekly publication.

The potential of gene therapy has long been hampered by the risks associated with using viruses as vectors to deliver healthy genes, but a new University of Georgia study helps bring scientists closer to a safe and efficient gene delivery method that doesn’t involve viruses.

Could injecting a gene into a patient with severe heart failure reverse their disabling and life-threatening condition? Physician-scientists are setting out to answer that question in a first-ever clinical trial of gene therapy to treat severe heart failure.

Replacing one amino acid on the surface of a virus that shepherds corrective genes into cells could be the breakthrough scientists have needed to make gene therapy a more viable option for treating genetic diseases such as hemophilia, University of Florida researchers say.

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.