Partial correction of cystinuria type A in mice via kidney-targeted transposon delivery

Partial correction of cystinuria type A in mice via kidney-targeted transposon delivery

We used kidney-targeted, non-viral, transposon-mediated gene delivery to express the mouse Slc3a1 transgene in one kidney of cystinuria type A (Slc3a1−/−) mice. We found a 44% reduction in urinary cystine concentration at 154 days post-gene transfer, although there was no significant effect on cysti...

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Bibliographic Details
Main Authors: Lauren E. Woodard, Richard C. Welch, Anna Menshikh, Wentian Luo, Felisha M. Williams, Jennifer L. Peek, Feng Sha, Ruth Ann Veach, Aparna Kaja, Thomas M. Beckerman, Talat Alp Ikizler, Matthew H. Wilson
Format: Article
Language:English
Published: Elsevier 2025-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
kidney
gene therapy
cystinuria
rBAT
Slc3a1
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125001933
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