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Article

Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases.

Cells - 29 Jan 2023

Hansen Silja, McClements Michelle E, Corydon Thomas J, MacLaren Robert E

Abstract excerpt

Inherited retinal diseases (IRD) are a clinically and genetically heterogenous group of diseases and a leading cause of blindness in the working-age population. Even though gene augmentation therapies have shown promising results, they are only feasible to treat a small number of autosomal recessive IRDs, because the size of the gene is limited by the vector used. DNA editing however could potentially correct...

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PMID: 36766782
DOI: 10.3390/cells12030440
PMCID: PMC9913839

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