Base editing-mediated splicing correction therapy for spinal muscular atrophy

查看全文 作　　者：Xiang [1,2]Lin;Haizhu [1]Chen;Ying-Qian [1]Lu;Shunyan [1]Hong;Xinde [3]Hu;Yanxia [3]Gao;Lu-Lu [1]Lai;Jin-Jing [1]Li;Zishuai [4]Wang;Wenqin [3]Ying;Lixiang [5]Ma;Ning [1,2]Wang;Erwei [4]Zuo;Hui [3]Yang;Wan-Jin [1,2]Chen

机构地区：[1]Deportment 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,Fudon University,Shanghai 200032,China

出　　处：《Cell Research》索引2020年第30卷第6期,共3页

基　　金：This work was supported by grants from the National Natural Science Foundation of China(81771230,31522037,U1905210,and 31922048);the Joint Funds for the Innovation of Science and Technology of Fujian Province(2017Y9094 and 2018Y9082);the National Key Clinical Specialty Discipline Construction Program,the Key Clinical Specialty Discipline Construction Program of Fujian,the National Science and Technology major project(2017YFC1001302);the Shanghai City Committee of Science and Technology project(16JC1420202);the Agricultural Science and Technology Innovation Program. 摘　　要：Dear Editor,Spinal muscular atrophy(SMA)is a devastating autosomal recessive motor neuron disease.1,2 Infants with more severe forms of type I SMA die before the age of 2 if no intervention is provided.1,2 Spinraza and zolgensma have been approved by the FDA as SMA therapeutics for pediatric patients.1,2 However,as an antisense oligonucleotide(ASO)based therapy,spinraza requires four loading doses,followed by three annual maintenance doses.The patients would be subjected to repeated intrathecal injections in the procedure.Zolgensma is a single-dose gene-replacement therapy for SMA,but unfortunately is unreliable in maintaining a high,stable level of gene expression.These issues have limited the therapeutic effects of the two approved drugs for SMA. 关键词：doses expression. ATROPHY

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Base editing-mediated splicing correction therapy for spinal muscular atrophy

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