Abstract: Provided herein are methods for the treatment of Alport Syndrome, using modified oligonucleotides targeted to miR-21. In certain embodiments, a modified oligonucleotide targeted to miR-21 improves kidney function and/or reduces fibrosis in subjects having Alport Syndrome. In certain embodiments, administration of a modified oligonucleotide targeted to miR-21 delays the onset of end-stage renal disease in a subject having Alport Syndrome. In certain embodiments, a modified oligonucleotide targeted to miR-21 delays the need for dialysis or kidney transplant in a subject having Alport Syndrome.
Type:
Grant
Filed:
May 10, 2016
Date of Patent:
June 27, 2017
Assignee:
Regulus Therapeutis Inc.
Inventors:
Jeremy Duffield, Balkrishen Bhat, Deidre MacKenna