Gene therapy has been used to target the mRNA (messenger RNA), a gene present in the mutated huntington gene (HTT). It has been discovered that a single administration of this therapy offers long-lasting medical benefits in Huntington’s disease. AAV, an adeno-associated virus gene therapy which contains a key artificial microRNA (pri-amiRNA) to target the mutated huntington gene mRNA has been portrayed in Human Gene Therapy, peer-reviewed journal.
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 Terence R. Flotte, MD, Editor-in-Chief of Human Gene Therapy
A methodical comparison was carried out by Dinah Sah, PhD, from Voyager Therapeutics, and co-authors, of pri-amiRNA processing of a number of AAV-pri-amiRNAs across several in vivo and in vitro, which included non-human primates and mice.
The researchers chose a tailored 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.“