Article

Exploring the Efficiency of Multi-Target Editing with Single Base Editors in Haematopoietic Cell lines

2026-01-16

Abstract excerpt

<title>Abstract</title> <p> Base editing, an extension of CRISPR-Cas9 gene editing, presents a promising strategy for correcting genetic mutations underlying inherited bone marrow failure syndromes. This approach enables <italic>ex vivo</italic> editing of haematopoietic stem cells, which can be reintroduced into patients without the risk of immune rejection. We have identified pathogenic single nucleotide mut...

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Literature Corpus work: a7e25d80-50b2-587c-bf56-2f89dd894ec5
DOI: 10.21203/rs.3.rs-8370034/v1

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