Non-viral ex vivo genome-editing in mouse bona fide hematopoietic stem cells with CRISPR/Cas9
Suvd Byambaa, Hideki Uosaki, Tsukasa Ohmori, Hiromasa Hara, Hitoshi Endo, Osamu Nureki, Yutaka Hanazono
Abstract
via non-homologous end-joining (NHEJ) with Cas9/RNP and a homology-independent targeted integration (HITI)-based construct. Repaired HSCs reconstituted T lymphocytes and thymuses in SCID mice. Our results show that a non-viral genome-editing of HSCs with CRISPR/Cas9 will help cure genetic diseases.
Topics & Concepts
CRISPRGenome editingHaematopoiesisEx vivoBiologyStem cellGenomeVirologyIn vivoGeneticsGeneCRISPR and Genetic EngineeringVirus-based gene therapy researchCAR-T cell therapy research