Correction of Fanconi Anemia Mutations Using Digital Genome Engineering

Correction of Fanconi Anemia Mutations Using Digital Genome Engineering

Fanconi anemia (FA) is a rare genetic disease in which genes essential for DNA repair are mutated. Both the interstrand crosslink (ICL) and double-strand break (DSB) repair pathways are disrupted in FA, leading to patient bone marrow failure (BMF) and cancer predisposition. The only curative therapy...

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Bibliographic Details
Main Authors: Christopher J. Sipe, Mitchell G. Kluesner, Samuel P. Bingea, Walker S. Lahr, Aneesha A. Andrew, Minjing Wang, Anthony P. DeFeo, Timothy L. Hinkel, Kanut Laoharawee, John E. Wagner, Margaret L. MacMillan, Gregory M. Vercellotti, Jakub Tolar, Mark J. Osborn, R. Scott McIvor, Beau R. Webber, Branden S. Moriarity
Format: Article
Language:English
Published: MDPI AG 2022-07-01
Series:International Journal of Molecular Sciences
Subjects:
Fanconi anemia (FA)
gene therapy
base editing
adenine base editing (ABE)
cytosine base editing (CBE)
CRISPR-Cas9
Online Access:https://www.mdpi.com/1422-0067/23/15/8416

Internet

https://www.mdpi.com/1422-0067/23/15/8416

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