Therapeutic Potential for CFTR Correctors in Autosomal Recessive Polycystic Kidney Disease

BACKGROUND & AIMS: Autosomal recessive polycystic kidney disease (ARPKD) is caused by mutations in PKHD1, encoding fibrocystin/polyductin (FPC). Severe disease occurs in perinates. Those who survive the neonatal period face a myriad of comorbidities, including systemic and portal hypertension, l...

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Detalles Bibliográficos
Autores principales: Yanda, Murali K., Tomar, Vartika, Cebotaru, Liudmila
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8529398/
https://www.ncbi.nlm.nih.gov/pubmed/34329764
http://dx.doi.org/10.1016/j.jcmgh.2021.07.012

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