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Article

Genetic treatment for autosomal dominant inherited retinal dystrophies: approaches, challenges and targeted genotypes.

The British journal of ophthalmology - 1 Sept 2023

Daich Varela Malena, Georgiadis Anastasios, Michaelides Michel

Abstract excerpt

Inherited retinal diseases (IRDs) have been in the front line of gene therapy development for the last decade, providing a useful platform to test novel therapeutic approaches. More than 40 clinical trials have been completed or are ongoing, tackling autosomal recessive and X-linked conditions, mostly through adeno-associated viral vector delivery of a normal copy of the disease-causing gene. However, only...

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PMID: 36038193
DOI: 10.1136/bjo-2022-321903

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