Curing genetic mutations for the next generation: CRISPR/ Cas9-mediated gene correction in mouse spermatogonial stem cells

• 来源期刊: 科学通报（英文版）
• 年，卷（期）: 2015,（3）
• 页码范围: 400-402
• 页数: 3页
• 语种: 中文
• DOI: 10.1007/s11434-015-0735-x

摘要

The CRISPR/Cas system is a very powerful and versatile tool for genomic manipulation.A recent study published in Cell Research by Wu et al.[1] reported a Cas9-mediated strategy to correct a disease-causing mutation in the mouse gammaC-crystallin(Crygc) locus in spermatogonial stem cells (SSCs) to cure the resulting cataracts disease in the offspring with 100 ％ efficiency.Importantly,no off-target mutations were identified through whole-genome sequencing.This study is a perfect combination of three different high biological technologies,SSCs manipulation,CRISPR/Cas gene editing and round spermatid injection (ROSI),shedding light on clinical applications for repair of genetic mutations in the progeny.