Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells
Author:
Affiliation:
1. Center for Medical Genetics, School of Life Sciences, Central South University, Hunan, China.
2. Hunan Jiahui Genetics Hospital, Changsha, Hunan, China.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2017.255
Reference45 articles.
1. Genetic risk assessment in carrier testing for spinal muscular atrophy
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3. Mutation update of spinal muscular atrophy in Spain: molecular characterization of 745 unrelated patients and identification of four novel mutations in the SMN1 gene
4. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy
5. An analysis of disease severity based on SMN2 copy number in adults with spinal muscular atrophy
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