Liu Lab Uses Prime Editing to Rescue an AHC Mouse Model

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.

These data provide the first 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 Dr. David Liu, Dr. Alex Sousa, and the Liu lab,?and from Dr. Cat Lutz and Dr. Markus Terrey, and their colleagues at The Jackson Laboratory.? 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.

Cure AHC, AHCF, and Hope for Annabel have worked together for many years to support this effort jointly, and it would not have been possible without enormous support from the Chan Zuckerberg Initiative. Many thanks to all involved for their massive effort.

The Voices of AHC (youtube.com)

Hope for Annabel - Annabel's Story 2019 (youtube.com)

Rare childhood disease prompts family to seek cure - The Washington Post