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Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

24 Feb 2021

Abstract excerpt

Lentivector gene therapy for X-linked chronic granulomatous disease (X-CGD) has proven to be a viable approach, but random vector integration and subnormal protein production from exogenous promoters in transduced cells remain concerning for long-term safety and efficacy. A previous genome editing-based approach using Streptococcus pyogenes Cas9 mRNA and an oligodeoxynucleotide donor to repair genetic mutations...

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PMID: 33623984
DOI: 10.1182/blood.2020008503

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