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 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, Ph.D., 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.
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