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A gene therapy for inherited blindness using dCas9-VPR–mediated transcriptional activation
Catalytically inactive dCas9 fused to transcriptional activators (dCas9-VPR) enables activation of silent genes. Many disease genes have counterparts, which serve similar functions but are expressed in distinct cell types. One attractive option to compensate for the missing function of a defective g...
Autores principales: | , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Association for the Advancement of Science
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7438099/ https://www.ncbi.nlm.nih.gov/pubmed/32875106 http://dx.doi.org/10.1126/sciadv.aba5614 |