ASGT Press Release 

Today's findings from the ASGT 6th Annual Meeting: New research on gene therapy of inherited and acquired diseases

New SCIDS treatment

(WASHINGTON, DC) - One of the most common forms of Severe Combined Immune Deficiency (SCID), also known as "bubble boy" disease, is Adenosine Deaminase (ADA) deficiency. A study presented at the 6th Annual Meeting of the American Society of Gene Therapy demonstrated a new approach to treating patients with ADA-SCID.

ADA-SCID is treated through a bone marrow transplant from the parent with the best immunologic match or by injections of purified ADA enzyme. However, a good match may not be available, the transplant not successful, or enzyme replacement therapy may not generate enough T and B cells. ADA-SCID has been shown to be a prime target for demonstrating the potential of gene therapy.

Alessandro Aiuti, MD and colleagues from the San Raffaele Telethon Institute for Gene Therapy, Italy; the University of Siena, Italy; the University of Turin, Italy; and the Hadassah University Hospital, Jerusalem exposed bone marrow calls from two ADA-SCID patients to retroviruses.

After treatment, enough ADA producing T and B cells emerged in these patients, resulting in them no longer needing the supplemental injection of purified ADA enzyme. The patients also did not experience any adverse effects or severe infections as a result of the therapy.

Treating muscular dystrophy

(WASHINGTON, DC) - A new gene transfer method was proven to be safe and effective in adding dystrophin to the muscles of patients with muscular dystrophy. These results were presented today at the 6th Annual Meeting of the American Society of Gene Therapy (ASGT).

Muscular dystrophy is caused by mutations in the dystrophin gene. Onset of the disease occurs before the age of four years and is characterized by a wasting away of the muscles, resulting in death after 20 years.

Norma B. Romero, MD, Institut de Myologie, Paris, France, and colleagues injected DNA containing the dystrophin gene into the radial muscle of nine patients with muscular dystrophy. The procedure was previously shown to be effective at transferring genes without any immune response in mice. The injections were not intended to treat the wasting away of all the muscles in the body, only specific sites. Muscle tissue was collected to determine whether the cells had accepted the DNA and generating dystrophin.

The researchers found dystorphin in the muscles of six of the nine patients, although muscle strength was not affected in any of the patients. In addition, no immune responses were detected. The study confirmed the procedure to be safe and effective. The results suggest that a more aggressive trial in which dystrophin DNA can be delivered to all muscle cells is in order.

New treatment for atopic dermatitis

(WASHINGTON, DC) - Japanese researchers have developed a topical ointment that effectively treats atopic dermatitis, according to a study presented today at the 6th Annual Meeting of the American Society of Gene Therapy (ASGT).

Atopic dermatitis, also known as eczema, is an incurable inflammation of the skin, often affecting the face, elbows and knees. The inflammation is caused by the production of inflammatory proteins such as Interleukins -1, -8, and 12, GM-CSF by cells in the inflamed region. The inflammatory genes require cells to make a protein called NFκB before the inflammatory genes become active.

Katsuto Tamai, MD, Odaka University School of Graduate Medicine, Japan, and colleagues treated seven patients with severe atopic dermatitis with an anti NFκB ointment containing a small piece of DNA that binds to the NFκB and prevents it from being translated into protein. After treatment with the ointment, the severity of the dermatitis improved in all seven patients, particularly in the face, with no adverse affects.

Currently, the only treatments for atopic dermatitis are corticosteroid creams, which are not effective in improving the quality of life in patients suffering from the condition. The success of the ointment may give new hope for sufferers of the debilitating condition.

The American Society of Gene Therapy is the largest medical professional organization representing researchers and scientists dedicated to discovering new gene therapies. ASGT was established in 1996, and has grown to more than 3,000 members. It is committed to promoting and fostering the general field of research involving gene therapy and to promoting professional and public education in all areas of gene therapy.