Donate with Us: Together Towards Treatment

Gene therapy has been changing lives for over three decades, but only for families facing conditions caused by a single gene. For chromosomal conditions like ours, where so many genes are affected and every 8p hero is wonderfully one of a kind, it has always felt out of reach.

Not anymore.

New techniques, called CRISPR activation and CRISPR inhibition, can turn genes up or down without cutting DNA. They're being used in humans for diseases caused by multiple genes, and have already shown they can restore brain activity in preclinical studies.

And we're ready for it, thanks to you. The results from the multi-omics study you funded in our spring campaign are here, and they're pointing us to the 8p genes that matter most. That gives us a roadmap for which genes to target with this innovative gene therapy approach.

Help us raise $70,000 by December 31

We have the backing of the scientists and researchers we need to adapt this approach for 8p, starting in the lab with cells from our heroes. All we need is the funds to take the next steps in bringing CRISPR activation and inhibition to our 8p genes.

If we reach our goal by the end of the year, we can launch this work in early 2027. Join us today and help us get there!

Employer Matching: Don’t forget to check if your employer offers matching contributions!

Every gift brings our 2027 start date closer. Give by December 31.

Together Towards Treatment