“Successful completion of this project will provide us with the necessary preclinical data and comprehensive strategy to bring CRISPR/Cas9-mediated gene correction closer to clinical application,” Dwi Kemaladewi said.
Dwi Kemaladewi, research associate in the Program of Genetics and Genome Biology at SickKids Research Institute, Toronto, Canada, was awarded an MDA development grant totaling $180,000 over three years to explore the potential of gene correction therapy in merosin-deficient congenital muscular dystrophy (MDC1A), which is caused by a mutation in the LAMA2 gene.
In studies conducted in an MDC1A mouse model, Kemaladewi and colleagues are working to determine whether CRISPR/Cas9 gene editing technology can restore LAMA2 gene expression and in turn, production of LAMA2 protein, an important component in the stability and organization of skeletal muscle and nerves.
If successful, Kemaladewi’s work could provide the necessary preclinical data and comprehensive strategy to bring CRISPR/Cas9-mediated gene correction closer to clinical application.
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