In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation

Development of efficacious in vivo delivery platforms for CRISPR-Cas9-based epigenome engineering will be critical to enable the ability to target human diseases without permanent modification of the genome. Toward this, we utilized split-Cas9 systems to develop a modular adeno-associated viral (AAV...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Moreno, Ana M., Fu, Xin, Zhu, Jie, Katrekar, Dhruva, Shih, Yu-Ru V., Marlett, John, Cabotaje, Jessica, Tat, Jasmine, Naughton, John, Lisowski, Leszek, Varghese, Shyni, Zhang, Kang, Mali, Prashant
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6035733/
https://ncbi.nlm.nih.gov/pubmed/29754775
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.04.017
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