Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells

Fanconi Anemia (FA) is a debilitating genetic disorder with a wide range of severe symptoms including bone marrow failure and predisposition to cancer. CRISPR-Cas genome editing manipulates genotypes by harnessing DNA repair and has been proposed as a potential cure for FA. But FA is caused by defic...

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Detalles Bibliográficos
Autores principales: Siegner, Sebastian M., Ugalde, Laura, Clemens, Alexandra, Garcia-Garcia, Laura, Bueren, Juan A., Rio, Paula, Karasu, Mehmet E., Corn, Jacob E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9653444/
https://www.ncbi.nlm.nih.gov/pubmed/36371486
http://dx.doi.org/10.1038/s41467-022-34479-z

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9653444/
https://www.ncbi.nlm.nih.gov/pubmed/36371486
http://dx.doi.org/10.1038/s41467-022-34479-z

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