In utero CRISPR-mediated therapeutic editing of metabolic genes

In utero gene editing has the potential to prenatally treat genetic diseases that result in significant morbidity and mortality before or shortly after birth. We assessed the viral vector-mediated delivery of clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated 9 (CRI...

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Detalles Bibliográficos
Autores principales: Rossidis, Avery C., Stratigis, John D., Chadwick, Alexandra C., Hartman, Heather A., Ahn, Nicholas J., Li, Haiying, Singh, Kshitiz, Coons, Barbara E., Li, Li, Lv, Wenjian, Zoltick, Philip W., Alapati, Deepthi, Zacharias, William, Jain, Rajan, Morrisey, Edward E., Musunuru, Kiran, Peranteau, William H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6249685/
https://www.ncbi.nlm.nih.gov/pubmed/30297903
http://dx.doi.org/10.1038/s41591-018-0184-6

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