Base editing-mediated splicing correction therapy for spinal muscular atrophy

Xiang Lin; Haizhu Chen; Ying-Qian Lu; Shunyan Hong; Xinde Hu; Yanxia Gao; Lu-Lu Lai; Jin-Jing Li; Zishuai Wang; Wenqin Ying; Lixiang Ma; Ning Wang; Erwei Zuo; Hui Yang; Wan-Jin Chen

doi:10.1038/s41422-020-0304-y

Base editing-mediated splicing correction therapy for spinal muscular atrophy

Cell Res. 2020 Jun;30(6):548-550. doi: 10.1038/s41422-020-0304-y. Epub 2020 Mar 24.

Authors

Xiang Lin^#^{1

2}, Haizhu Chen^#¹, Ying-Qian Lu^#¹, Shunyan Hong^#¹, Xinde Hu^#³, Yanxia Gao³, Lu-Lu Lai¹, Jin-Jing Li¹, Zishuai Wang⁴, Wenqin Ying³, Lixiang Ma⁵, Ning Wang^{1

2}, Erwei Zuo⁶, Hui Yang⁷, Wan-Jin Chen^{8

9}

Affiliations

¹ Department of Neurology and Institute of Neurology, First Affiliated Hospital, Institute of Neuroscience, Fujian Medical University, Fuzhou, 350005, China.
² Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, 350005, China.
³ Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, 200031, China.
⁴ Lingnan Guangdong Laboratory of Modern Agriculture, Genome Analysis Laboratory of the Ministry of Agriculture, Agricultural Genomics Institute at Shenzhen, Chinese Academy of Agricultural Sciences, Shenzhen, 518124, China.
⁵ Department of Anatomy, Histology & Embryology, Shanghai Medical College, Fudan University, Shanghai, 200032, China.
⁶ Lingnan Guangdong Laboratory of Modern Agriculture, Genome Analysis Laboratory of the Ministry of Agriculture, Agricultural Genomics Institute at Shenzhen, Chinese Academy of Agricultural Sciences, Shenzhen, 518124, China. erweizuo@163.com.
⁷ Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, 200031, China. huiyang@ion.ac.cn.
⁸ Department of Neurology and Institute of Neurology, First Affiliated Hospital, Institute of Neuroscience, Fujian Medical University, Fuzhou, 350005, China. wanjinchen75@fjmu.edu.cn.
⁹ Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, 350005, China. wanjinchen75@fjmu.edu.cn.

^# Contributed equally.

No abstract available

Publication types

Letter
Research Support, Non-U.S. Gov't

MeSH terms

Animals
Genetic Therapy*
HEK293 Cells
Humans
Induced Pluripotent Stem Cells
Mice
Mice, Knockout
Mouse Embryonic Stem Cells
Muscular Atrophy, Spinal / therapy*
RNA Editing
Survival of Motor Neuron 1 Protein / genetics*
Survival of Motor Neuron 2 Protein / genetics

Substances

SMN1 protein, human
SMN2 protein, human
Smn1 protein, mouse
Survival of Motor Neuron 1 Protein
Survival of Motor Neuron 2 Protein