gene therapy

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. 

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.

A new study provides evidence that methods using human bone marrow-derived stem cells to deliver gene therapy to cure diseases of the blood, bone marrow and certain types of cancer do not cause the development of tumors or leukemia.

Researchers have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness -- Leber congenital amaurosis.

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.

For some inherited diseases, one barrier to successful gene therapy is that a commonly used vector (package into which the curative gene is placed) cannot accommodate the large size of the curative gene.

However, a newly developed vector derived from the AAV5 form of adeno-associated virus has been used to successfully carry large genes into cells and to improve eye function in a mouse model of an inherited disease causing progressive loss of sight.

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.

Earlier research had similar findings for alcohol intake. Treatments that increase levels of these chemicals -- dopamine D2 receptors -- may prove useful in treating addiction.

A novel strategy for circumventing safety problems that have plagued gene therapy has been offered by a complex multicenter group of researchers.

The study reports that adenovirus type 5, a common vector for delivering gene therapy, transfects liver cells by a different mechanism than previously thought.

That mechanism offers a new target for modifying the viral vector to make it safe for clinical use.