A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

Adeno-associated viral (AAV) vectors packaging the CRISPR-Cas9 system (AAV-CRISPR) can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV vectors are a preferred delivery vehicle for tissue-directed gene therapy because of their ability to achieve sustained expres...

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Dades bibliogràfiques
Publicat a:Mol Ther Methods Clin Dev
Autors principals: Li, Ang, Lee, Ciaran M., Hurley, Ayrea E., Jarrett, Kelsey E., De Giorgi, Marco, Lu, Weiqi, Balderrama, Karol S., Doerfler, Alexandria M., Deshmukh, Harshavardhan, Ray, Anirban, Bao, Gang, Lagor, William R.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2018
Matèries:
Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6313841/
https://ncbi.nlm.nih.gov/pubmed/30619914
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.11.009
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