Gene Correction Therapy Study, Seed-Funded by Cure CMD, Continues through MDA Grant


What: A $180,000 grant has just been announced by the Muscular Dystrophy Association (MDA) to fund the exploration of potential of gene correction therapy in merosin-deficient congenital muscular dystrophy (MDC1A), which is caused by mutations in the LAMA2 gene. If successful, this work could provide the necessary preclinical data and comprehensive strategy to bring CRISPR/Cas9-mediated gene correction closer to clinical application.

For several years, Cure CMD seeded this project with a $100,000 grant award to the Cohn Lab, in addition to providing support for the improvement of the LAMA2 mouse model, critical to this research. Funding of early research such as this is what sets Cure CMD apart from other, larger funding organizations. With help from the CMD community, Cure CMD pioneers early research that can lead to significant breakthroughs and draw awareness from other potential funders.

Who: Dr. Dwi Kemaladewi, research associate in the Program of Genetics and Genome Biology at SickKids Research Institute in Toronto, Canada, along with Dr. Ronni Cohn, Senior Scientist at the same Institute and Chief of Pediatrics at the Hospital for Sick Children.

When: The $180,000 grant from the MDA will be awarded over three years, until 2021. Cure CMD’s previous support of Drs. Kemaladewi and Cohn occurred from May 2015 to May 2017 (www.curecmd.org/funding-portfolio).

Where: Research will be conducted at the SickKids Research Institute in Toronto, Canada.

Why: Research of the CMDs is severely underfunded. As Cure CMD is the only U.S. patient advocacy organization fully dedicated to supporting research in LAMA2-CMD and improving the quality of life for those living with it, the organization and its supporters are the pioneers for CMD research Cure CMD

Cure CMD was founded in 2008 to advance research for treatments and a cure for congenital muscular dystrophy (CMD) and, through engagement and support of the CMD community, to also improve the lives of those living with CMD. To date, the organization has funded more than $2 million in research, launched an international patient registry with more than 2,500 registrants, assisted with genetic diagnosis, and connected more than 2,000 affected individuals and their families to a supportive, helpful community.

#LAMA2 #MDC1A #CRISPR #Kemaladewi #Cohn #SickKids #CureCMD #ACureIsAmongUs

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