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...

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Detalles Bibliográficos
Autores principales: Böhm, Sybille, Splith, Victoria, Riedmayr, Lisa Maria, Rötzer, René Dominik, Gasparoni, Gilles, Nordström, Karl J. V., Wagner, Johanna Elisabeth, Hinrichsmeyer, Klara Sonnie, Walter, Jörn, Wahl-Schott, Christian, Fenske, Stefanie, Biel, Martin, Michalakis, Stylianos, Becirovic, Elvir
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2020
Materias:
Research Articles
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

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