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Dear Friends and Families of the AHC Patient Community:

We want to update you on some exciting news related to our gene editing project.

Over the last several months, the Liu Lab has tested their gene editing strategy in AHC mice at the Jackson Laboratory. Preliminary results are encouraging.

The Liu team has found that treatment with prime editors can correct the Atp1a3 D801N mutation in the mouse brain and improve multiple clinically-relevant symptoms in D801N mice.

This is the first reported in vivo rescue of a neurological disease with prime editing.

This data is the first piece of evidence that prime editing may work as a therapeutic strategy in vivo in AHC. We are still many steps away from a therapy for AHC patients, but this result is a very positive first step on the long road ahead.

These findings are also the result of many years of effort from Drs. David Liu, Alex Sousa, and the Liu lab, and from Drs. Cat Lutz and Markus Terrey, and their colleagues at Jax. We are truly grateful to partner with such an incredible team.

Future experiments designed to edit the E815K mutation in mice will begin soon. The Liu team has made history. They have taken AHC to the front lines of cutting-edge experimental research.

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