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Effective CRISPR/Cas9-mediated correction of a Fanconi anemia defect by error-prone end joining or templated repair

Abstract:

Fanconi anemia (FA) is a cancer predisposition syndrome characterized by congenital abnormalities, bone marrow failure, and hypersensitivity to aldehydes and crosslinking agents. For FA patients, gene editing holds promise for therapeutic applications aimed at functionally restoring mutated genes in hematopoietic stem cells. However, intrinsic FA DNA repair defects may obstruct gene editing feasibility. Here, we report on the CRISPR/Cas9-mediated correction of a disruptive mutation in Fancf. ...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's Version

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Publisher copy:
10.1038/s41598-018-36506-w

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Role:
Author
ORCID:
0000-0001-7486-5291
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Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Pathology Dunn School
Role:
Author
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Publisher:
Springer Nature Publisher's website
Journal:
Scientific Reports Journal website
Volume:
9
Pages:
Article: 768
Publication date:
2019-01-25
Acceptance date:
2018-11-19
DOI:
EISSN:
2045-2322
ISSN:
2045-2322
Pubs id:
pubs:969164
URN:
uri:d153c16a-cd68-4323-81c7-d5b1d887c7af
UUID:
uuid:d153c16a-cd68-4323-81c7-d5b1d887c7af
Local pid:
pubs:969164
Language:
English

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