Preclinical evaluation of AAV9-coSMN1 gene therapy for spinal muscular atrophy: efficacy and safety in mouse models and non-human primates

Preclinical evaluation of AAV9-coSMN1 gene therapy for spinal muscular atrophy: efficacy and safety in mouse models and non-human primates

Abstract Background Spinal muscular atrophy (SMA) is a severe neuromuscular disorder caused by the loss of motor neurons in the spinal cord. Our team has initiated clinical trials using adeno-associated virus serotype 9 (AAV9) vectors carrying a codon-optimized human SMN1 (coSMN1) gene, delivered vi...

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Main Authors: Wenhao Ma, Zhijie Wu, Tianyi Zhao, Yan Xia, Jing Qin, Xue Tian, Xin Li, Jun He, Yan Zhang, Lina Zhang, Li Li, Zheyue Dong, Zhichun Feng, Xiaoyan Dong, Wang Sheng, Xiaobing Wu
Format: Article
Language:English
Published: BMC 2025-04-01
Series:Molecular Medicine
Online Access:https://doi.org/10.1186/s10020-025-01207-4
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https://doi.org/10.1186/s10020-025-01207-4

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