Article

<i>CTNS</i> mRNA as a potential treatment for nephropathic cystinosis

2023-04-29

Abstract excerpt

Messenger RNA (mRNA) therapies are emerging in different disease areas, but have not yet reached the kidney field. Our aim was to study the feasibility to correct the genetic defect in nephropathic cystinosis using synthetic mRNA. Cystinosis is a prototype disorder of proximal tubular dysfunction caused by mutations in the CTNS gene, encoding the lysosomal cystine-H + symporter cystinosin, and leading to cystine...

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Literature Corpus work: 61a6ebe7-e943-5952-a328-38bfd907ac3b
DOI: 10.1101/2023.04.28.538106

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