Strength is More than Muscle®
and we are proud to share our strength with Cure Rare Disease in efforts to develop Treatment for those living with Duchenne Muscular Dystrophy.
Fritz and Monkel Lek, lead researcher with CRD
Drug development for rare disease is challenging. The “one size fits all” approach to drug development is not sufficient, and we are choosing to take another shot on goal in the rare disease space with a technology called CRISPR.
For patients with Duchenne muscular dystrophy, CRISPR technology offers one of the greatest opportunities to restore the dystrophin protein to muscles by editing the DMD gene. Short for clusters of regularly interspaced short palindromic repeats, CRISPR relies on a complex immune response bacteria use to fend off viruses by cutting their DNA.
Researchers discovered how to harness this mechanism by programming CRISPR to act as “molecular scissors,” deleting or replacing strands of mutated DNA in order to modify gene function. In the case of Duchenne, it can be used to restore the ability of the DMD gene to produce dystrophin. This has already been proven in animal models.
Duchenne Research Update:
April 2021 Fritz had a muscle biopsy. This biopsy is used to set up both a cell line and develop a mouse model with Fritz’s specific DMD causing mutation.
Jan-July 2022, CRISPR constructs were developed and tested on his cell line, showing efficacy. A
Sep-Feb 2022, A mouse model containing Fritz’s specific DMD causing mutation was developed. A colony was engineered, characterized, and prepped for further safety & efficacy experiments.
Sept 2023- Dec 2023 mice and materials are being prepped for the 12 wk pharm-tox study to measure efficacy of this therapeutic.
*This is a specific update on Fritz’s project, however there are many other boys who are also benefiting in similar ways from the strength you share!
