Gene editing could replace a lifetime of medication, but there's a catch

Imagine receiving a single treatment that could keep your cholesterol under control for decades, potentially eliminating the need for daily medication. Advances in gene editing are bringing that possibility closer to reality. But scientists say the same technology that could revolutionize medicine also raises difficult questions about safety, permanent genetic changes, and unintended consequences.

Gene therapy is already being used to treat certain serious illnesses. These treatments can introduce working copies of genes, silence harmful genetic activity, or make changes directly to DNA. Unlike conventional medications that must be taken repeatedly, some genetic treatments are designed to work after just one administration.

More than 6,000 rare genetic disorders have been identified, although only a small proportion currently have targets that can be treated. Gene therapies have already achieved important successes against conditions such as spinal muscular atrophy and sickle cell disease. Now, researchers are exploring whether similar approaches could help prevent or manage far more widespread health problems, including high cholesterol, heart disease, obesity, and diabetes.

That raises a fundamental question: Just because scientists can permanently change a person's genetic activity, does that mean they should? In a perspective published in JAMA Internal Medicine, University of Michigan Health Frankel Cardiovascular Center cardiologists Eric D. Smith, M.D., and Venkatesh L.

Murthy, M.D., Ph.D., outline how doctors and researchers should weigh the potential benefits of genomic therapies against their risks. The two gene therapy researchers explain why these treatments are so promising, what makes them different from conventional medicines, and what must happen before they can be safely offered to millions of people. Why One-Time Gene Therapy Is Different From Daily Medication One of the most appealing features of gene therapy is the possibility of achieving lasting results without repeated treatments.

Traditional medicines generally work only as long as patients continue taking them. Genomic therapies operate differently because they act on genetic material itself. They may provide a healthy version of a gene, suppress a gene responsible for harmful effects, or modify a specific DNA sequence.