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

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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Bibliografische gegevens
Hoofdauteurs: Manish Kumar Yadav, Kyung Whan Yoo, Anthony Atala, Baisong Lu
Formaat: Artikel
Taal:English
Gepubliceerd in: Elsevier 2022-12-01
Reeks:Molecular Therapy: Methods & Clinical Development
Onderwerpen:
CLCN5
megalin
CRISPR-Cas9
gene therapy
lentiviral vector
immune response
Online toegang:http://www.sciencedirect.com/science/article/pii/S2329050122001346

Internet

http://www.sciencedirect.com/science/article/pii/S2329050122001346

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