CRISPR vs cholesterol: can gene editing prevent heart disease?

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Musunuru says he knows of about a dozen companies that intend to use gene editing to tackle high cholesterol. Other cardiovascular projects include treatments for a rarer condition called transthyretin amyloidosis, which can lead to heart failure. Biotechnology company CRISPR Therapeutics in Zug, Switzerland — the sponsor of the current cholesterol trial — has begun work on a gene-editing therapy to treat high blood pressure. “It’s fertile ground,” says Musunuru, who is himself a co-founder of a company that’s pursuing similar treatments.
But shifting from rare diseases to more common ones will require big safety studies that are capable of detecting unusual side effects, and CRISPR-based therapies have been used in only a few hundred people so far.