SAN DIEGO – In the past 20 years, gene therapy has advanced from concept to effective clinical application, according to a presentation at the American Society of Gene Therapy’s 12th Annual Meeting, May 27.
Adenosine deficiency disorder, a condition known commonly as bubble boy disease, has been the focus of several recent international, long-term trials of gene therapy and is the treatment’s most significant success story to date. By correcting the defective genes that cause ADA deficiency, and grafting those repaired cells to patients, researchers have engineered a reliable cure.
The advancements in gene therapy that made treatment of ADA deficiency possible may soon translate to other conditions.
“The technologies and understanding gained through gene therapy for ADA deficiency will have benefits for other inherited diseases of the hematopoietic and immune systems,” said Bobby Gaspar, MD, PhD, Professor of Pediatrics and Immunology at the Institute of Child Health in London. “We are already seeing successes in other immunodeficiencies, such as Wiskott-Aldrich syndrome.”
ADA deficiency is rare, but its impact is extreme. Patients, usually diagnosed in the first six months of life, suffer a defective immune system, their bodies unable to defend against even minor infections and viruses. They are susceptible to serious and recurrent illness.
ADA deficiency was the first condition treated by gene therapy, beginning in the 1990s. Initial trials showed only modest success but, today, more than 20 patients worldwide have been cured.
The American Society of Gene Therapy (ASGT) 12th Annual Meeting is the world’s largest scientific meeting surrounding the latest developments in gene and cell therapy, attended by nearly 2,000 researchers from around the world and featuring 60 scientific presentations.
ASGT is a non-profit medical and professional organization representing researchers and scientists devoted to the discovery of new gene and cell therapies. Established in 1996, ASGT is the largest association of individuals involved in gene and cell therapeutics with approximately 2,500 members in the United States and worldwide. Additional information is available at www.asgt.org.
Contact: Kimberly Jahnke (kjahnke@execinc.com)
ASGT Executive Office: 414.278.1341
ASGT Press Room (May 27-30): 619.525.6286
Cell: 414.803.3746
