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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...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2019
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| Materias: | |
| 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 |