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1. (WO2018204788) METHODS FOR TREATMENT OF ALPORT SYNDROME

Pub. No.:    WO/2018/204788    International Application No.:    PCT/US2018/031094
Publication Date: Fri Nov 09 00:59:59 CET 2018 International Filing Date: Sat May 05 01:59:59 CEST 2018
IPC: A61K 31/7125
A61P 13/12
C12N 15/113
Applicants: REGULUS THERAPEUTICS INC.
Inventors: WRIGHT, Timothy
Title: METHODS FOR TREATMENT OF ALPORT SYNDROME
Abstract:
Provided herein are methods for the treatment of Alport syndrome, using a modified oligonucleotide targeted to miR-21. In certain embodiments, the modified oligonucleotide targeted to miR-21 improves kidney function and/or reduces fibrosis in subjects having Alport syndrome. In certain embodiments, administration of the modified oligonucleotide targeted to miR-21 delays the onset of end-stage renal disease in a subject having Alport syndrome. In certain embodiments, the modified oligonucleotide targeted to miR-21 delays the need for dialysis or kidney transplant in a subject having Alport syndrome.