Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease

Autosomal dominant polycystic kidney disease (ADPKD), caused by mutations in either PKD1 or PKD2 genes, is one of the most common human monogenetic disorders and the leading genetic cause of end-stage renal disease. Unfortunately, treatment options for ADPKD are limited. Here we report the discovery...

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Detalles Bibliográficos
Autores principales: Lee, Edmund C., Valencia, Tania, Allerson, Charles, Schairer, Annelie, Flaten, Andrea, Yheskel, Matanel, Kersjes, Kara, Li, Jian, Gatto, Sole, Takhar, Mandeep, Lockton, Steven, Pavlicek, Adam, Kim, Michael, Chu, Tiffany, Soriano, Randy, Davis, Scott, Androsavich, John R., Sarwary, Salma, Owen, Tate, Kaplan, Julia, Liu, Kai, Jang, Graham, Neben, Steven, Bentley, Philip, Wright, Timothy, Patel, Vishal
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6742637/
https://www.ncbi.nlm.nih.gov/pubmed/31515477
http://dx.doi.org/10.1038/s41467-019-11918-y

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