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A Potential Therapy Using Antisense Oligonucleotides to Treat Autosomal Recessive Polycystic Kidney Disease

(1) Background: Autosomal recessive polycystic kidney disease (ARPKD) is a rare ciliopathy characterized by progressively enlarged kidneys with fusiform dilatation of the collecting ducts. Loss-of-function mutations in the PKHD1 gene, which encodes fibrocystin/polyductin, cause ARPKD; however, an ef...

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Detalles Bibliográficos
Autores principales: Li, Huixia, Wang, Chunli, Che, Ruochen, Zheng, Bixia, Zhou, Wei, Huang, Songming, Jia, Zhanjun, Zhang, Aihua, Zhao, Fei, Ding, Guixia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9966971/
https://www.ncbi.nlm.nih.gov/pubmed/36835961
http://dx.doi.org/10.3390/jcm12041428