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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...
Autores principales: | Marrocco, Elena, Maritato, Rosa, Botta, Salvatore, Esposito, Marianna, Surace, Enrico Maria |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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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 |
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