Gene Therapy for the Treatment of Huntington’s Disease

MARY ANN LIEBERT, INC./GENETIC ENGINEERING NEWS

IMAGE: THE FIRST PEER-REVIEWED JOURNAL IN THE FIELD AND PROVIDES ALL-INCLUSIVE ACCESS TO THE CRITICAL PILLARS OF HUMAN GENE THERAPY: RESEARCH, METHODS, AND CLINICAL APPLICATIONS.

CREDIT: MARY ANN LIEBERT INC., PUBLISHERS

Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit in Huntington’s disease after a single administration. An adeno-associated virus (AAV) gene therapy containing a primary artificial microRNA (pri-amiRNA) targeting HTT mRNA is described in the peer-reviewed journal Human Gene Therapy. Click here to read the article now.

“The challenge of treating autosomal dominant genetic brain disorders has been very daunting, and this pioneering effort by Voyager Therapeutics provides new hope for patients with one of the most tragic such diagnoses, that of Huntington’s disease,” says Editor-in-Chief of Human Gene Therapy Terence R. Flotte, MD. 

Dinah Sah, PhD, from Voyager Therapeutics, and coauthors conducted a systematic comparison of pri-amiRNA processing of a number of AAV-pri-amiRNAs across multiple in vitro and in vivo systems, including mice and non-human primates.

The investigators selected an optimized pri-amiRNA “for an AAV gene therapy that exhibits efficient and precise pri-amiRNA processing, along with potent pharmacological activity for HTT lowering and general tolerability in vivo for the treatment of Huntington’s disease.”

About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available on the Human Gene Therapy website.