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Lead investigator

Research Sites & Affiliations

• University of Lübeck, Lübeck, Germany

Funding partners: Muscular Dystrophy Canada ,   Cure CMD

Budget: $80,000 USD

Disorders: Collagen 6-related dystrophy (Bethlem and Ullrich congenital muscular dystrophies)

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

In recent years, there has been rapid development of gene therapy tools in genetic disorders. With this project the researchers are taking the first of many steps towards establishing a potential therapy using the novel CRISPRoff technology to target dominant glycine changes causing COL6-RD.

Impact: