Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology
Huntington disease (HD) is a fatal, neurodegenerative genetic disorder with aggregation of mutant Huntingtin protein (mutHTT) in the brain as a key pathological mechanism.There are currently no disease modifying therapies for HD; however, HTT-lowering therapies hold promise.Recombinant adeno-associated virus serotype 5 expressing a microRNA that ta