ASGCT Press Release

ASGCT 13th Annual Meeting:
Gene Therapy Reverses Muscle Weakness in MD Patients

WASHINGTON – Gene therapy may restore muscle control in patients with certain forms of muscular dystrophy, according to research to be presented at the American Society of Gene & Cell Therapy Annual Meeting, May 17-22.

In a small study, patients with limb-girdle muscular dystrophy received a transplant of healthy genes. After six months, a majority of patients showed increased levels of muscle fiber and gene expression similar to those in healthy patients with normal muscle control.

“This marks significant progress in the study of gene therapy for muscular dystrophy,” said lead researcher Jerry Mendell, MD. “This is the first successful gene therapy that resulted in gene expression for such a long period.”

Mendell will present his findings at 2:15 p.m. Friday, May 21.

Members of the media interested in attending the ASGCT Annual Meeting should download the press registration form at www.asgct.org/am10.

Limb-girdle muscular dystrophy is a progressive disease characterized by loss of strength in the muscles around the hips and shoulders. LGMD occurs when certain genes do not produce an adequate amount of muscle proteins. As muscles weaken, patients lose the ability to control their arms or legs and are often confined to a scooter or wheelchair. There are currently no reliable, successful treatments to reverse damage done by the disease.

This study lays the foundation for expanded research. Scientists next hope to deliver the gene to the femoral artery, directly targeting patients’ leg muscles.

Abstract: 290
Title: Sustained Alpha-Sarcoglycan Gene Expression in LGMD2D Following Gene Transfer
Date: Friday, May 21, 2010
Session Info: Simultaneous Oral Abstract Sessions: Clinical Gene & Cell Therapy Oral Abstract Session (2 - 4 p.m.)
Presentation Time: 2:15 p.m.
Room: Marriott Ballroom, Salons 2 & 3

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, www.asgct.org.

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