Article

Gene surgery as a potential treatment option for Nephropathic Cystinosis <i>in vitro</i>

2023-11-03

Abstract excerpt

Nephropathic cystinosis is a rare monogenetic kidney disease caused by mutations in the lysosomal transporter cystinosin (encoded by CTNS ) that, to date, has no cure. The hallmark of this disease is lysosomal accumulation of cystine and decline in proximal tubular function leading to kidney failure early in life. In this project, we developed a novel gene repair strategy using CRISPR/Cas9 Homology-Independent Ta...

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Literature Corpus work: 20fc203f-78e4-5918-a6e2-a8686e5e0dfc
DOI: 10.1101/2023.11.01.565117

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