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Challenging Safety and Efficacy of Retinal Gene Therapies by Retinogenesis

Gene-expression programs modulated by transcription factors (TFs) mediate key developmental events. Here, we show that the synthetic transcriptional repressor (TR; ZF6-DB), designed to treat Rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), does not perturb murine retinal develo...

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Detalles Bibliográficos
Autores principales: Marrocco, Elena, Maritato, Rosa, Botta, Salvatore, Esposito, Marianna, Surace, Enrico Maria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8198227/
https://www.ncbi.nlm.nih.gov/pubmed/34071252
http://dx.doi.org/10.3390/ijms22115767