Welcome to Behind the Episode, a rare disease advocacy podcast by Cure AHC. Months ago, we gathered your questions as a community — and this conversation is where those answers live.
This is a longer episode, because we didn’t want to cut any of the information that feels so important and so hopeful for families. But we also know your time is limited, so we’ve added chapters and time stamps in the show notes so you can easily jump to the sections you need.
The recent prime-editing success in the AHC mouse model is the first time a neurological or central nervous system disease has ever been successfully genetically edited in a living animal.
Not in a dish.
Not in isolated cells.
In a living mammal.
We’ve seen CRISPR breakthroughs in the eye, the liver, heart and blood — you may have heard about Baby KJ, or the FDA-approved treatments for sickle cell disease and metabolic liver disorders…
But crossing the blood–brain barrier and successfully editing a neurological disease in vivo has never happened before.
Until now.
You’ll find links in the show notes to the key action steps from today’s episode and more information about guests Nina and Simon Frost, Rare Hope and other relevant resources.
