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Article

CRISPR medicine for blood disorders: Progress and challenges in delivery

6 Jan 2023

Abstract excerpt

Blood disorders are a group of diseases including hematological neoplasms, clotting disorders and orphan immune deficiency diseases that affects human health. Current improvements in genome editing based therapeutics demonstrated preclinical and clinical proof to treat different blood disorders. Genome editing components such as Cas nucleases, guide RNAs and base editors are supplied in the form of either a...

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Identifiers

PMID: 36687779
DOI: 10.3389/fgeed.2022.1037290

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