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Jul 30, 2023

Genome editing prevents hypertrophic cardiomyopathy in mice

Posted by in categories: biotech/medical, genetics

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.

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