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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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Backstrom, Jon R., Sheng, Jinsong, Wang, Michael C., Bernardo-Colón, Alexandra, Rex, Tonia S.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7530248/
https://ncbi.nlm.nih.gov/pubmed/33024795
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.09.001
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