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Molecular Therapies for Inherited Retinal Diseases—Current Standing, Opportunities and Challenges

Inherited retinal diseases (IRDs) are both genetically and clinically highly heterogeneous and have long been considered incurable. Following the successful development of a gene augmentation therapy for biallelic RPE65-associated IRD, this view has changed. As a result, many different therapeutic a...

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Bibliographic Details
Published in:Genes (Basel)
Main Authors: Vázquez-Domínguez, Irene, Garanto, Alejandro, Collin, Rob W. J.
Format: Artigo
Language:Inglês
Published: MDPI 2019
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6770110/
https://ncbi.nlm.nih.gov/pubmed/31466352
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/genes10090654
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