Gene Therapy for Treatment of Huntington’s Disease (HD) Shows Signs of Benefit in Pre-Clinical Studies

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. The January 17, 2022 open-access article is titled “Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy In Vitro and in Mice and Nonhuman Primates.”