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Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease
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Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease
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Journal Article
Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease
2019
Overview
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 and characterization of RGLS4326, a first-in-class, short oligonucleotide inhibitor of microRNA-17 (miR-17), as a potential treatment for ADPKD. RGLS4326 is discovered by screening a chemically diverse and rationally designed library of anti-miR-17 oligonucleotides for optimal pharmaceutical properties. RGLS4326 preferentially distributes to kidney and collecting duct-derived cysts, displaces miR-17 from translationally active polysomes, and de-represses multiple miR-17 mRNA targets including
Pkd1
and
Pkd2
. Importantly, RGLS4326 demonstrates a favorable preclinical safety profile and attenuates cyst growth in human in vitro ADPKD models and multiple PKD mouse models after subcutaneous administration. The preclinical characteristics of RGLS4326 support its clinical development as a disease-modifying treatment for ADPKD.
Autosomal dominant polycystic kidney disease (ADPKD) is a leading genetic cause of end-stage renal disease with limited treatment options. Here the authors discover and characterize a microRNA inhibitor as a potential treatment for ADPKD.
Publisher
Nature Publishing Group UK,Nature Publishing Group,Nature Portfolio
Subject
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/ 59
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/ Animals
/ Cell Proliferation - drug effects
/ Cysts
/ Gene Regulatory Networks - drug effects
/ Hematopoiesis - drug effects
/ Humanities and Social Sciences
/ Humans
/ Kidneys
/ Male
/ MicroRNAs - antagonists & inhibitors
/ miRNA
/ mRNA
/ Mutation
/ Oligonucleotides - pharmacokinetics
/ Oligonucleotides - pharmacology
/ Oligonucleotides - therapeutic use
/ Polycystic Kidney Diseases - drug therapy
/ Polycystic Kidney Diseases - genetics
/ RNA
/ Science
/ Sodium
