Fine-tuning FAM161A gene augmentation therapy to restore retinal function

Fine-tuning FAM161A gene augmentation therapy to restore retinal function

Abstract For 15 years, gene therapy has been viewed as a beacon of hope for inherited retinal diseases. Many preclinical investigations have centered around vectors with maximal gene expression capabilities, yet despite efficient gene transfer, minimal physiological improvements have been observed i...

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Bibliographic Details
Main Authors: Yvan Arsenijevic, Ning Chang, Olivier Mercey, Younes El Fersioui, Hanna Koskiniemi-Kuendig, Caroline Joubert, Alexis-Pierre Bemelmans, Carlo Rivolta, Eyal Banin, Dror Sharon, Paul Guichard, Virginie Hamel, Corinne Kostic
Format: Article
Language:English
Published: Springer Nature 2024-03-01
Series:EMBO Molecular Medicine
Subjects:
Ciliopathy
Gene Therapy
Promoter Activity
Retinal Degeneration
Online Access:https://doi.org/10.1038/s44321-024-00053-x

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