ACC Holds Its First Roundtable on Gene Editing Therapy
More than 94 million adults in the U.S. live with high cholesterol, and thousands are affected by transthyretin amyloidosis (ATTR), a progressive and often underdiagnosed condition. The ACC recently hosted a Heart House Roundtable addressing a new approach to these conditions: gene editing. The roundtable discussed how gene editing can silence or correct disease-driving genes, such as TTR in ATTR and PCSK9 in hyperlipidemia, offering alternatives to lifelong medication and mitigating progressive disease. "Gene editing is already demonstrating remarkable results in early trials, but major questions remain," said ACC President Christopher Kramer, MD, FACC. "How can we ensure safety and long-term efficacy? What are the clinical criteria for selecting eligible patients? How do we handle the ethical considerations of germline cell changes or off-target effects? And perhaps most important, how do we ensure equitable access to these potentially life-altering therapies?" Read the key takeaways.