Xiaowen Shen

TL;DR: A homology-mediated end joining (HMEJ)-based strategy is devised, using CRISPR/Cas9-mediated cleavage of both transgene donor vector that contains guide RNA target sites and ∼800 bp of homology arms, and the targeted genome, with an efficiency much greater than HR-, NHEJ- and MMEJ-based strategies.

TL;DR: A transgenic mouse is generated using an improved dCas9 system that enables simultaneous and precise in vivo transcriptional activation of multiple genes and long noncoding RNAs in the nervous system and is able to use targeted activation of endogenous neurogenic genes in these transgenic mice to directly and efficiently convert astrocytes into functional neurons in vivo.

TL;DR: A microhomology-mediated end-joining (MMEJ)-based strategy for precisely targeted gene integration in transfected neurons and hepatocytes in vivo with efficiencies up to 20%, much higher than HDR-based strategy in adult mouse tissues.

TL;DR: This study provides proof-of-principle for a new strategy to therapeutically intervene in SMA and other RNA-splicing-related diseases.

TL;DR: In this article, the authors developed a generalizable endogenous transcription-gated switch that releases single-guide RNAs in the presence of an endogenous promoter, which provides a powerful platform to sense the activity of endogenous genetic elements underlying cellular functions.