CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells

CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells

Fanconi anemia (FA) is an inherited condition characterized by impaired DNA repair, physical anomalies, bone marrow failure, and increased incidence of malignancy. Gene editing holds great potential to precisely correct the underlying genetic cause such that gene expression remains under the endogen...

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Bibliographic Details
Main Authors: Karolina Skvarova Kramarzova, Mark J. Osborn, Beau R. Webber, Anthony P. DeFeo, Amber N. McElroy, Chong Jai Kim, Jakub Tolar
Format: Article
Language:English
Published: MDPI AG 2017-06-01
Series:International Journal of Molecular Sciences
Subjects:
gene editing
CRISPR/Cas9
Fanconi anemia
fibroblasts
Fanconi anemia D1
poly adenosine diphosphate-ribose polymerase inhibitors
Online Access:http://www.mdpi.com/1422-0067/18/6/1269

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http://www.mdpi.com/1422-0067/18/6/1269

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