Tech Comm Available Technologies :: The Research Institute at Nationwide Children's Hospital, Columbus, Ohio


Project TitleAAV Delivery of Mir29 to Suppress Fibrosis in Muscular Dystrophy
Track Code2016-007
Short Description

Researchers at Nationwide Children's Hospital have developed an a deno-associated virus (AAV) approach to deliver MicroRNA 29 (miR-29) to suppress fibrosis in Duchenne Muscular Dystrophy (DMD). 


Duchenne muscular dystrophy (DMD) is caused by dystrophin deficiency resulting in muscle loss and progressive muscle weakness and fibrotic scarring. Fibrotic infiltration is profound in DMD and is a significant impediment to any potential therapy. Researchers at Nationwide Children's Hospital have addressed this problem using an adeno-associated virus (AAV) approach to deliver MicroRNA 29 (miR-29) to suppress fibrosis. Initial results using AAV-delivered miR-29 as an anti-fibrotic therapy suggest that there is significant beneficial effect with reduced collagen and elastin levels, which are key contributors in fibrosis. This work provides a rationale for overexpression of miR-29 to reduce fibrosis along with dystrophin replacement in translational, pre-clinical studies.

TagsDMD, Duchenne muscular dystrophy, dystrophin, AAV, microRNA, fibrosis, anti-fibrotic, therapy, therapeutic, scarring
Posted DateSep 25, 2017 3:23 PM


Jerry Mendell
Louise Rodino-Klapac
Kristin Heller


Margaret Barkett


Intellectual Property

Patent Number Issue Date Type Country of Filing
None None Other Patent None
Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000