Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

Abstract Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec (Zolgensma®) is an approved adeno-associated virus (AAV) vector gene therapy for infants with spinal muscular atrophy (SMA), however, adverse events have been observed in both ani...

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Bibliographic Details
Main Authors: Qing Xie, Xiupeng Chen, Hong Ma, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Gerald F Cox, Fiona Weaver, Jun Yang, Zachary Kennedy, Alisha Gruntman, Ailing Du, Qin Su, Ran He, Phillip WL Tai, Guangping Gao, Jun Xie
Format: Article
Language:English
Published: Springer Nature 2024-02-01
Series:EMBO Molecular Medicine
Subjects:
AAV
SMA
SMN1
Gene Therapy
Endogenous Promoter
Online Access:https://doi.org/10.1038/s44321-024-00037-x

Internet

https://doi.org/10.1038/s44321-024-00037-x

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