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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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Detalhes bibliográficos
Publicado no:Int J Mol Sci
Main Authors: Skvarova Kramarzova, Karolina, Osborn, Mark J., Webber, Beau R., DeFeo, Anthony P., McElroy, Amber N., Kim, Chong Jai, Tolar, Jakub
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5486091/
https://ncbi.nlm.nih.gov/pubmed/28613254
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms18061269
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