Could Gene Editing Eventually Offer a ‘One-Time’ Treatment for CVD?

Cardiovascular diseases could be among the world’s first medical conditions to be treated by changing a patient’s genes. If ongoing phase 3 trials succeed, gene editing will offer a novel therapy for transthyretin amyloidosis, and treatments for hyperlipidemia may not be far behind. 

Precise gene editing using clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) was discovered in 2012. The discovery triggered worldwide efforts to apply the technology to correct gene-based diseases. But, to date, the FDA and the European Medicines Agency have approved only one gene editing therapy, for sickle cell disease.