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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Detalhes bibliográficos
Publicado no:Sci Adv
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: American Association for the Advancement of Science 2020
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7438099/
https://ncbi.nlm.nih.gov/pubmed/32875106
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aba5614
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