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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...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2021
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Materias: | |
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 |