Thanks to the work of Cure Rare Disease, and their partnership with Charles River, a humanized mouse model, or what we like to call “Fritz mice," containing the humanized dystrophin gene with Fritz’s particular mutation, was developed and bred! This particular mouse has taken over a year + tens of thousands of dollars to breed. Your donations over the course of 2021 helped fund this crucial, and very complicated, step of drug development! Now that we have mice with Fritz's mutation, we can move forward with testing a specific CRISPR based therapeutic on these mice, in hopes of correcting the mutation. This next step is crucial for testing both safety and efficacy of the therapeutic.
Fritz Mouse Model Developed!