Article

Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene Therapy

2023-02-23

Abstract excerpt

On-target toxicity to normal cells is a major safety concern with targeted immune and gene therapies. Here, we developed a base editing (BE) approach exploiting a naturally occurring CD33 single nucleotide polymorphism leading to removal of full-length CD33 surface expression on edited cells. CD33 editing in human and nonhuman primate (NHP) hematopoietic stem and progenitor cells (HSPCs) protects from CD33-targete...

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Literature Corpus work: f46baac0-7aa3-5e5b-8c19-a301529fefaa
DOI: 10.1101/2023.02.23.529353

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