Optimization of S. aureus dCas9 and CRISPRi Elements for a Single Adeno-Associated Virus that Targets an Endogenous Gene

The power of CRISPRi to decrease targeted gene expression for clinical applications has been inhibited by delivery challenges. Existing constructs are too large to fit within the ∼4.7 kb packaging size limitation of adeno-associated virus (AAV), the only FDA approved viral vector for clinical use. T...

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Detalles Bibliográficos
Autores principales: Backstrom, Jon R., Sheng, Jinsong, Wang, Michael C., Bernardo-Colón, Alexandra, Rex, Tonia S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7530248/
https://www.ncbi.nlm.nih.gov/pubmed/33024795
http://dx.doi.org/10.1016/j.omtm.2020.09.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7530248/
https://www.ncbi.nlm.nih.gov/pubmed/33024795
http://dx.doi.org/10.1016/j.omtm.2020.09.001

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