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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Publicat a:Nat Med
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: 2018
Matèries:
Article
Accés en línia: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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