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Base and Prime Editing in the Retina—From Preclinical Research toward Human Clinical Trials

16 Oct 2022

Abstract excerpt

Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of diseases that are one of the leading causes of vision loss in young and aged individuals. IRDs are mainly caused by a loss of the post-mitotic photoreceptor neurons of the retina, or by the degeneration of the retinal pigment epithelium. Unfortunately, once these cells are damaged, it is irreversible and leads to permanent...

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PMID: 36293232
DOI: 10.3390/ijms232012375

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