Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

In vivo tissue-specific genome editing at the desired loci is still a challenge. Here, we report that AAV9-delivery of truncated guide RNAs (gRNAs) and Cas9 under the control of a computationally designed hepatocyte-specific promoter lead to liver-specific and sequence-specific targeting in the mous...

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Detalles Bibliográficos
Publicado en:Mol Ther
Main Authors: Singh, Kshitiz, Evens, Hanneke, Nair, Nisha, Rincón, Melvin Y., Sarcar, Shilpita, Samara-Kuko, Ermira, Chuah, Marinee K., VandenDriessche, Thierry
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2018
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5993986/
https://ncbi.nlm.nih.gov/pubmed/29599079
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.02.023
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