Translation of the CRISPR–Cas9 system to human therapeutics holds high promise. However,
specificity remains a concern especially when modifying stem cell populations. We show …

Immune checkpoint therapy has resulted in remarkable improvements in the outcome for
certain cancers. To broaden the clinical impact of checkpoint targeting, we devised a strategy …

Though AsCas12a fills a crucial gap in the current genome editing toolbox, it exhibits relatively
poor editing efficiency, restricting its overall utility. Here we isolate an engineered variant, …

Genome editing using the CRISPR/Cas9 system requires the presence of guide RNAs bound
to the Cas9 endonuclease as a ribonucleoprotein (RNP) complex in cells, which cleaves …

CRISPR–Cas proteins are RNA-guided nucleases used to introduce double-stranded breaks
(DSBs) at targeted genomic loci. DSBs are repaired by endogenous cellular pathways …

We report the generation of mice with an intact and functional copy of the 2.3-megabase human
dystrophin gene (hDMD), the largest functional stretch of human DNA thus far integrated …

Cobblestone (type II) lissencephaly and mental retardation are characteristic features of a
subset of congenital muscular dystrophies that include Walker–Warburg syndrome, muscle-…

Chimeric antigen receptor (CAR) redirected T cells are potent therapeutic options against
hematological malignancies. The current dominant manufacturing approach for CAR T cells …

Genome editing of human cluster of differentiation 34 + (CD34 + ) hematopoietic stem and
progenitor cells (HSPCs) holds great therapeutic potential. This study aimed to optimize on-…

T-cell malignancies are associated with frequent relapse and high morbidity, which is partly
due to the lack of effective or targeted treatment options. To broaden the use of CAR-T cells …