Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene
时间控制和肌肉特异性 CRISPR/Cas9 介导的 DMPK 基因中 CTG 重复扩增的删除
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2021.11.024
Beatrice Cardinali, Claudia Provenzano, Mariapaola Izzo, Christine Voellenkle, Jonathan Battistini, Georgios Strimpakos, Elisabetta Golini, Silvia Mandillo, Ferdinando Scavizzi, Marcello Raspa, Alessandra Perfetti, Denisa Baci, Dejan Lazarevic, Jose Manuel Garcia-Manteiga, Geneviève Gourdon, Fabio M