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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Publicado no:Nat Med
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6249685/
https://ncbi.nlm.nih.gov/pubmed/30297903
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41591-018-0184-6
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