CRISPR Campaign 2025
Life-changing and historic gene therapies are within reach! Cure KCNH1 Foundation is an Encinitas nonprofit working with local biotech companies and national and international organizations and researchers to advance cutting-edge treatments that can help not only Tristan and the other KCNH1 kids, but millions of other rare-disease patients. Please watch our video to learn more about KCNH1 and our family's journey, and donate today to make a difference in the lives of these severely-disabled kids!​​
Dear Cure KCNH1 Supporters,
We are facing an urgent deadline to advance our CRISPR gene-editing project. As you may know from following our progress over the past year, we won a $50K grant last winter to fund this promising initiative at Northwestern University, in collaboration with leading rare-disease researcher, Dr. Al George. You may have seen all over the news this past spring, the story of Baby KJ, who has a rare liver disease and was cured with a CRISPR gene therapy. This is precisely the type of treatment that Dr. George is working on for KCNH1, and unlike many of the other types of treatments that we're working on (which nonetheless still hold promise for helping our kids!), this approach represents a true cure for KCNH1 mutations by actually fixing that one incorrect nucleotide (out of 3.2 billion!) that messes everything up for our KCNH1 kids.
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Science is complicated and usually prone to delays and setbacks, but this project has moved incredibly quickly and smoothly. We've all been kind of blown away by the impressive results, including Dr. George - I've NEVER seen him so enthusiastic and effusive about a project! Dr. George is ready to initiate the final phase of experiments, which involves testing the gene editors in our recently-completed KCNH1 mouse model. This is an extremely exciting milestone, since we have been working on these mouse models for several years now, and this will be the first time we get to use them to test out one of our many promising treatments. If the gene editors successfully treat the symptoms of the KCNH1 mice, Dr. George will be in a position to pursue larger grants to continue the development of this therapeutic and hopefully bring it all the way to clinical trials.
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The organization that granted us the initial $50K has committed to an additional $50K matching grant (we raise $50K, they give us $50K) to fund this next phase of the gene-editing project. We have $15K left to unlock the other $50K, and we need to get this to Dr. George within the next two weeks to keep the experiments on track. This is truly our opportunity to cure KCNH1, so please join us in supporting this transformative project!
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In gratitude,
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Michaelle Jinnette
Founder and President
Cure KCNH1 Foundation
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