Article

Adenine base editing is an efficient approach to restore function in FA patient cells without double-stranded DNA breaks

2022-04-22

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 deficiencies in DNA repair itself, preventing the use of editing strategies such as homology directed repair. Recently develo...

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Literature Corpus work: 31eb01d9-fcb1-5c4e-86e7-a265aa6b3099
DOI: 10.1101/2022.04.22.489197

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