Low-dose AAV9-SMN1 with CNS-selective expression delivers efficacy and favorable safety in spinal muscular atrophy

Molecular Therapy 2026 Open access

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Authors: Yongguo Yu, Xiji Qin, Mengxia Jing, Carol Huang, Xin Zhou, Yanjie Fan, Jia Rao, Hongping Xia, Weiqian Dai, Xiaomei Luo, Hui Liu, Shihua Cao
+8 more Yanjun Shen, Yao Qiu, Qianru Zhang, Shuqi Li, Ling Su, Yi Wang (32470), Jay Hou, Kun Sun
DOI: 10.1016/j.ymthe.2026.02.050
Cited by: 1
References: 31

Fields of Study & Subjects

Neurogenetic and Muscular Disorders Research Muscle Physiology and Disorders Viral Infections and Immunology Research

Publication details

Journal / source
Molecular Therapy
Publisher
Elsevier BV
Volume
34
Issue
6
Pages
3287–3299
Publication date
4 March 2026
Language
EN