Victor Lin

TL;DR: ObLiGaRe is described, a new method for site-specific gene insertions that uses the efficient NHEJ pathway and acts independently of HR, and has enabled us to insert a 15-kb inducible gene expression cassette at a defined locus in human cell lines.

TL;DR: In vivo pharmacological inhibition of mTOR is sufficient to normalize aberrant autophagy and improve muscle phenotypes in Mtm1 null mice, suggesting that aberrant mTORC1 signaling and impairedAutophagy are consequences of the loss of MTM1 and may play a primary role in disease pathogenesis.

TL;DR: Trans-membrane 203 is an evolutionarily conserved regulator of cellular calcium homeostasis, is required for spermatogenesis and provides a causal link between intracellular calcium regulation and spermiogenesis.

TL;DR: To evaluate whether gene editing alters the self-renewal, engraftment or lineage differentiation capacity of HSCs, gene-edited HSPCs were transplanted into NOD- scid IL2rγnull mice to assess reconstitution of the hematopoietic system and data support the development of autologous genome-modified HSPC transplantation as a novel cell therapy for SCD.