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Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells

12 Nov 2022

Abstract excerpt

Fanconi Anemia (FA) is a debilitating genetic disorder with a wide range of severe symptoms including bone marrow failure and predisposition to cancer. CRISPR-Cas genome editing manipulates genotypes by harnessing DNA repair and has been proposed as a potential cure for FA. But FA is caused by deficiencies in DNA repair itself, preventing the use of editing strategies such as homology directed repair. Recently...

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PMID: 36371486
DOI: 10.1038/s41467-022-34479-z

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