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Regulus Presents Positive Preclinical Data Demonstrating that microRNA-21 Plays an Important Role in Alport Syndrome
Date:11/8/2013

ser toward the selection of an anti-miR-21 clinical development candidate and we remain excited about the potential to bring this innovative treatment to patients with this devastating disease." 

Additionally, microRNA therapeutics will be discussed in two oral presentations at the meeting:

  • On Friday, November 8, 2013 at 2:00 p.m. EST, Jeremy Duffield M.D., Ph.D., Regulus' collaborator at the University of Washington, will give an oral presentation titled "MicroRNAs Are Novel Therapeutic Targets to Treat Kidney Injury and Fibrosis"; and  
  • On Saturday, November 9, 2013 at 5:42 p.m. EST, Regulus scientists and representatives from its collaborator at the University of Washington will give an oral presentation titled "Anti-miR21 Protects Collagen 4A3 Deficient Mice from Progression of Alport Disease by Decreasing Oxidative Stress"

About miR-21 and Alport Syndrome

According to the National Institutes of Health, Alport Syndrome occurs in approximately 1 in 50,000 newborns.  Alport syndrome is a genetic condition characterized by kidney disease, hearing loss, and eye abnormalities.  The kidneys become less able to function as this condition progresses, resulting in end-stage renal disease (ESRD).

Mutations in the COL4A3, COL4A4, and COL4A5 genes cause Alport Syndrome. These genes provide instructions for making one component of a protein called type IV collagen. This protein plays an important role in the kidneys, specifically in structures called glomeruli. Glomeruli are clusters of specialized blood vessels that remove water and waste products from blood and create urine. Mutations in these genes result in abnormalities of the type IV collagen in glomeruli, which prevents the kidneys from properly filtering the blood and allows blood and protein to pass into the urine.

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SOURCE Regulus Therapeutics Inc.
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