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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...
| Autores principales: | , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
MDPI
2017
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5486091/ https://www.ncbi.nlm.nih.gov/pubmed/28613254 http://dx.doi.org/10.3390/ijms18061269 |