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Terence R. Flotte, MD, and Christian Mueller, PhD, discuss their recent findings related to recombinant adeno-associated viruses as a delivery system for gene therapy, which were recently published in the Journal of Clinical Investigation.

experts interviewed on progress of gene therapy trials Tay-Sachs experts interviewed on progress of gene therapy trials Miguel Sena-Esteves talks about the promise of clinical trials Jim Fessenden UMass Medical School Communications Miguel Sena-Esteves, PhD, associate professor of neurology and a leading force behind the creation of the Tay-Sachs Gene Therapy Consortium, an international collaboration of scientists committed to translating current gene therapy results into a human clinical trial

UMass Medical School has entered into a three-year, collaborative research agreement with Pfizer to evaluate determinants that influence the manufacturing quality and yield of viral vectors used in gene therapy. The research at UMMS is being performed under the direction of Guangping Gao, PhD, and Dan Wang, PhD.

Researchers at the University of Massachusetts Amherst and UMass Chan Medical School mapped the expression and maturation of the protein alpha-1 antitrypsin (AAT) with unprecedented clarity. UMass Chan gene therapy pioneer Terence R. Flotte, MD, is a co-author on the paper.

Almost two decades after gene therapy was hailed as a potential cure for a myriad of diseases, scientists at UMass Medical School are leading a resurgence in treatment strategies that have had some notable successes and are attracting investors and the public, according to a front page story in the Boston Sunday Globe.

Data from a study by Terence Flotte and UMass Medical School colleagues demonstrates sustained protein expression 5 years after a gene therapy for AAT deficiency.

Flotte named associate editor of Human Gene Therapy- UMass Medical School - Worcester Flotte named associate editor of Human Gene Therapy Ellie Castano UMass Medical School Communications Terence R. Flotte, MD, executive deputy chancellor and provost, dean of the School of Medicine, the Celia and Isaac Haidak Professor in Medicine and professor of pediatrics, has been named an associate editor of the journal Human Gene Therapy, which publishes scientific papers on original investigations into the

Spark Therapeutics and the UMMS Horae Gene Therapy Center have entered into a research collaboration to develop next generation viral vectors for gene therapy.

Gene therapy and CRISPR strategies for curing blindness Gene therapy and CRISPR strategies for curing blindness Researchers are now testing treatments for several kinds of visual impairment Hemant Khanna, PhD, for The Conversation For The Conversation In a piece originally published on The Conversation, Hemant Khanna, PhD, comments on recent studies that use CRISPR to treat diseases that cause blindness and explains why the eye is an ideal organ for testing new therapeutic approaches. In a piece

Top story: Boston toddler Noa Greenwood, who was one of the first to receive an investigational gene therapy for Canavan disease developed at UMass Chan Medical School, delivered a very special “thank you” to the research team in a visit with her parents earlier this year.