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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Detalles Bibliográficos
Autores principales: McDougald, Devin S., Duong, Thu T., Palozola, Katherine C., Marsh, Anson, Papp, Tyler E., Mills, Jason A., Zhou, Shangzhen, Bennett, Jean
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6477656/
https://www.ncbi.nlm.nih.gov/pubmed/31024980
http://dx.doi.org/10.1016/j.omtm.2019.03.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6477656/
https://www.ncbi.nlm.nih.gov/pubmed/31024980
http://dx.doi.org/10.1016/j.omtm.2019.03.004

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