Article

Preventing vision loss in a mouse model of Leber Congenital Amaurosis by engineered tRNA

2025-07-11

Abstract excerpt

Premature termination codons (PTCs) are associated with rare genetic disorders. Inducing targeted read-through of these ‘nonsense mutations’ presents a potential therapeutic strategy for modifying disease outcomes. We previously reported that one such PTC, W53X, in the KCNJ13 gene causes blindness and Leber congenital amaurosis type-16 (LCA-16) due to loss of function of the inwardly rectifying potassium channel...

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Literature Corpus work: b92bceac-924c-5b76-8915-70e189c79c4a
DOI: 10.1101/2025.07.10.660754

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