Lifeboat Foundation

In a recent study published in the journal Nature Medicine, researchers pursued one-time cures for hypertrophic cardiomyopathy (HCM). They used a previously constructed murine model of HCM, designated as R403Q-129SvEv, to evaluate two different genetic therapies, as follows:

I) an adenine base editor (ABE8e)

Ii) a potent Cas9 nuclease delivered by an adeno-associated virus (AAV) vector.