Flotte, Mueller discuss gene therapy advance

By Bryan Goodchild and Mark L. Shelton

UMass Medical School Communications

November 18, 2013

Recombinant adeno-associated viruses as a delivery system for gene therapy have shown promise for the treatment of diseases like alpha-1 antitrypsin deficiency, a chronic lung disorder: however, it’s generally believed that the immune system would eventually eliminate the gene-expressing cells, causing a loss of effectiveness or requiring subsequent retreatment.

In a new paper in the Journal of Clinical Investigation, Terence R. Flotte, MD, the Celia and Isaac Haidak Professor of Medical Education and professor of pediatrics and microbiology & physiological systems, and Christian Mueller, PhD, assistant professor of pediatrics, and colleagues have shown persistent gene expression for a period of more than a year in a Phase II gene therapy human trial, providing additional support for gene therapy as a therapeutic tool.

The paper, “Year-long rAAV-mediated transgene expression associated with Treg responses in humans” is the subject of a video interview with Drs. Flotte and Mueller at the JCI website.