- 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. ...
Expand abstract - Publication status:
- Published
- Peer review status:
- Peer reviewed
- Version:
- Publisher's Version
- Files:
-
-
(pdf, 2.4MB)
-
- Publisher copy:
- 10.1038/s41598-018-36506-w
-
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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:
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2045-2322
- ISSN:
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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
- Copyright holder:
- Van De Vrugt et al.
- Copyright date:
- 2019
- Notes:
- © The Author(s) 2019. This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. Te images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
Journal article
Effective CRISPR/Cas9-mediated correction of a Fanconi anemia defect by error-prone end joining or templated repair
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