Nature Communications (Sep 2019)

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

  • Edmund C. Lee,
  • Tania Valencia,
  • Charles Allerson,
  • Annelie Schairer,
  • Andrea Flaten,
  • Matanel Yheskel,
  • Kara Kersjes,
  • Jian Li,
  • Sole Gatto,
  • Mandeep Takhar,
  • Steven Lockton,
  • Adam Pavlicek,
  • Michael Kim,
  • Tiffany Chu,
  • Randy Soriano,
  • Scott Davis,
  • John R. Androsavich,
  • Salma Sarwary,
  • Tate Owen,
  • Julia Kaplan,
  • Kai Liu,
  • Graham Jang,
  • Steven Neben,
  • Philip Bentley,
  • Timothy Wright,
  • Vishal Patel

DOI
https://doi.org/10.1038/s41467-019-11918-y
Journal volume & issue
Vol. 10, no. 1
pp. 1 – 14

Abstract

Read online

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.