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Lentiviral vector mediated gene therapy for type I Dent disease ameliorates Dent disease-like phenotypes for three months in ClC-5 null mice

Type 1 Dent disease is caused by changes in chloride voltage-gated channel 5 (CLCN5) gene on chromosome X, which causes the lack or dysfunction of chloride channel ClC-5. Affected subjects show proteinuria and hypercalciuria, and eventually develop end-stage kidney disease. Currently there is no cur...

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Detalles Bibliográficos
Autores principales: Yadav, Manish Kumar, Yoo, Kyung Whan, Atala, Anthony, Lu, Baisong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9574595/
https://www.ncbi.nlm.nih.gov/pubmed/36284768
http://dx.doi.org/10.1016/j.omtm.2022.09.009