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Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene Therapy
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Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene Therapy
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Journal Article
Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene Therapy
2023
Overview
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-targeted therapeutics without affecting normal hematopoiesis
, thus demonstrating potential for novel immunotherapies with reduced off-leukemia toxicity. For broader applications to gene therapies, we demonstrated highly efficient (>70%) multiplexed adenine base editing of the CD33 and gamma globin genes, resulting in long-term persistence of dual gene-edited cells with HbF reactivation in NHPs.
, dual gene-edited cells could be enriched via treatment with the CD33 antibody-drug conjugate, gemtuzumab ozogamicin (GO). Together, our results highlight the potential of adenine base editors for improved immune and gene therapies.
Publisher
Cold Spring Harbor Laboratory
Subject
