Challenging Safety and Efficacy of Retinal Gene Therapies by Retinogenesis

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...

Full description

Bibliographic Details
Main Authors: Elena Marrocco, Rosa Maritato, Salvatore Botta, Marianna Esposito, Enrico Maria Surace
Format: Article
Language:English
Published: MDPI AG 2021-05-01
Series:International Journal of Molecular Sciences
Subjects:
gene therapy
adeno-associated virus (AAV)
retinal degeneration
transcription
zinc finger
retinitis pigmentosa
Online Access:https://www.mdpi.com/1422-0067/22/11/5767

Internet

https://www.mdpi.com/1422-0067/22/11/5767

Similar Items