Gene editing could replace a lifetime of medication, but there's a catch
A single gene-editing treatment could someday protect against heart disease, high cholesterol, and other common conditions for decades, potentially replacing lifelong medications. But scientists warn that permanently altering genes introduces risks that could take years to emerge, making long-term safety a critical challenge.
Key points
- Gene editing could offer one-time treatments for conditions like high cholesterol, heart disease, obesity and diabetes, but permanent genetic changes raise safety questions.
- Eric D. Smith and Venkatesh L. Murthy of University of Michigan Health propose five factors for weighing the benefits and risks of genomic therapies.
- Some existing gene therapies have list prices above $1 million per patient, raising concerns about affordability and access.
Advances in gene editing could one day deliver a single treatment that controls cholesterol for decades and removes the need for daily medication, but scientists say the same technology raises hard questions about permanent genetic changes and unintended consequences. Cardiologists Eric D. Smith, M.D., and Venkatesh L. Murthy, M.D., Ph.D., of University of Michigan Health outline how doctors and researchers should weigh these benefits against the risks in a perspective published in JAMA Internal Medicine.
Gene therapy is already used for certain serious illnesses and has achieved important successes against conditions such as spinal muscular atrophy and sickle cell disease. More than 6,000 rare genetic disorders have been identified, though only a small proportion currently have targets that can be treated. Researchers are now exploring whether similar approaches could prevent widespread problems including high cholesterol, heart disease, obesity and diabetes.
Why one-time treatment differs
Conventional medicines work only while patients keep taking them and can be adjusted or stopped if side effects appear. Genomic therapies act on genetic material itself, making treatments intended to work after a single dose possible. However, many gene therapies use modified viruses to carry genetic instructions into cells, and the immune system can recognize these delivery vehicles, making repeat administration difficult or impossible even if benefits fade. Current therapies generally target somatic cells and are not intended to create inherited changes.
Just because scientists can permanently change a person's genetic activity, does that mean they should?
— the article
Safety, monitoring and cost
Murthy notes that a rare serious side effect could still harm many people if a therapy reaches millions, so treatments for widespread use must meet especially demanding safety standards. Smith argues long-term monitoring cannot be an afterthought and will require independent, standardized systems operating at a large scale. The authors also flag cost: some existing gene therapies have list prices above $1 million per patient, and applying similar pricing to common diseases would strain healthcare systems and could worsen inequalities.
Early cholesterol studies
Researchers are already conducting early studies into whether a single genetic treatment can produce lasting reductions in LDL, or bad, cholesterol, which contributes to artery buildup and cardiovascular risk. The authors say promising early results will not be enough; before routine use, researchers need evidence that benefits last, risks stay low over many years, costs are manageable and reliable safeguards exist.
