CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters

Validation of gene transfer vectors containing tissue-specific promoters in cell-based functional assays poses a formidable challenge for gene therapy product development. Here, we describe a novel approach based on CRISPR/dCas9 transcriptional activation to achieve robust transgene expression from...

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Udgivet i:Mol Ther Methods Clin Dev
Main Authors: McDougald, Devin S., Duong, Thu T., Palozola, Katherine C., Marsh, Anson, Papp, Tyler E., Mills, Jason A., Zhou, Shangzhen, Bennett, Jean
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6477656/
https://ncbi.nlm.nih.gov/pubmed/31024980
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.03.004
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