Seamless genetic conversion of SMN2 to SMN1 via CRISPR/Cpf1 and single-stranded oligodeoxynucleotides in spinal muscular atrophy patient-specific iPSCs
Zhou, Miaojin, Hu, Zhiqing, Qiu, Liyan, Zhou, Tao, Feng, Mai, Hu, Qian, Zeng, Baitao, Li, Zhuo, Sun, Qianru, Wu, Yong, Liu, Xionghao, Wu, Lingqian, Liang, DeshengLanguage:
english
Journal:
Human Gene Therapy
DOI:
10.1089/hum.2017.255
Date:
March, 2018
File:
PDF, 7.33 MB
english, 2018