Commentary: Current Status of Gene Therapy for Spinal Muscular Atrophy.

As a caveat, since both SMA patients died within few months of the onasemnogene treatment, findings may be representative of unsuccessful cases of gene therapy and may not represent typical distribution and efficacy of onasemnogene. Onasemnogene (Synonyms: Onasemnogene abeparvovec, Zolgensma, AVXS-101), an adeno-associated virus serotype 9 (AAV9)-mediated gene therapy was approved in 2019 for the treatment of SMA (Al-Zaidy et al., [1]). Keywords: spinal muscular atrophy (SMA); Survival Motor Neuron (SMN); onasemnogene abeparvovec; Zolgensma; AVXS-101; gene therapy; AAV9 EN spinal muscular atrophy (SMA) Survival Motor Neuron (SMN) onasemnogene abeparvovec Zolgensma AVXS-101 gene therapy AAV9 1 4 4 05/19/22 20220517 NES 220517 Introduction Spinal muscular atrophy (SMA) is a genetic disease of a broad spectrum of severity, ranging from infant mortality to adult onset (Singh et al., [9]). Spinal muscular atrophy (SMA), Survival Motor Neuron (SMN), onasemnogene abeparvovec, gene therapy, Zolgensma, AVXS-101, AAV9. [Extracted from the article]