Jerry R. Mendell

Jerry Mendell is a pioneer in gene therapy for neuromuscular diseases who led development of Zolgensma (onasemnogene abeparvovec), the first AAV gene therapy approved for spinal muscular atrophy. His work has demonstrated that single-dose gene therapy can halt disease progression and transform outcomes for genetic diseases.

Jerry Mendell是神经肌肉疾病基因治疗的先驱，领导了Zolgensma（onasemnogene abeparvovec）的开发，这是第一个获批用于脊髓性肌萎缩症的AAV基因治疗。他的工作表明单剂量基因治疗可以阻止疾病进展并改变遗传疾病的结果。