George Yohrling of the HDSA Explains AMT-130 Gene Therapy – Rare Disease Report
Rare Disease Report |
George Yohrling of the HDSA Explains AMT-130 Gene Therapy
Rare Disease Report Yohrling: The AMT-130 is a gene therapy, and it's a Huntingtin-lowering approach that would be mediated through viral delivery of adeno-associated virus, or AAV. In the case of uniQure, they use AAV-5, which will deliver a micro-RNA, and this micro-RNA … |
