An amiRNA based on pri-miR-451 and delivered via an AAV5 vector (AMT-130) was demonstrated to be safe and efficient for allele-nonselective silencing of huntingtin in a few animal models of HD.17, 18, 19 A phase I/IIa clinical trial (NCT0412049) was started this year to investigate the safety and persistence of AMT-130 in the brain. Here, HTT is linked to Huntington disease.