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Effective CRISPR/Cas9-mediated correction of a Fanconi anemia defect by error-prone end joining or templated repair
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...
Autores principales: | van de Vrugt, Henri J., Harmsen, Tim, Riepsaame, Joey, Alexantya, Georgina, van Mil, Saskia E., de Vries, Yne, Bin Ali, Rahmen, Huijbers, Ivo J., Dorsman, Josephine C., Wolthuis, Rob M. F., te Riele, Hein |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6347620/ https://www.ncbi.nlm.nih.gov/pubmed/30683899 http://dx.doi.org/10.1038/s41598-018-36506-w |
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