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A Curative DNA Code for Hematopoietic Defects

27 Jun 2022

Abstract excerpt

Innovations in programmable nucleases have expanded genetic engineering capabilities, raising the possibility of a new approach to curing monogenic hematological diseases. Feasibility studies using ex vivo targeted genome-editing, and nonintegrating viral vectors show outstanding potential for correcting genetic conditions at their root cause. This article reviews the latest technological advances in the...

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PMID: 35773054
DOI: 10.1016/j.hoc.2022.05.002

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