Article

Exploring Prime Editing strategies for genetic correction of a Ceroid Neuronal Lipofuscinosis type 2 disease causing variant

2025-10-03

Abstract excerpt

<h4>ABSTRACT</h4> Neuronal ceroid lipofuscinosis type 2 (CLN2) is a rare, fatal paediatric neurodegenerative genetic disease that causes progressive psychomotor decline, epilepsy, speech impairment, vision loss, and premature death by late childhood or early adolescence. It is caused by mutations in the TPP1 gene, which encodes the tripeptidyl peptidase 1 enzyme. To date, enzyme replacement therapy (ERT) with r...

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Literature Corpus work: 1643dde0-08fe-500c-b410-90144fd206cb
DOI: 10.1101/2025.10.02.680174

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