Should Gene Therapy Be Used for Common Diseases? Two Cardiologists Propose Five Questions
Gene therapy began with rare, severe diseases such as spinal muscular atrophy and sickle cell disease. Now gene editing is moving toward some of the most common conditions in medicine, including high cholesterol and heart disease. In a perspective published in October 2026 in JAMA Internal Medicine, two University of Michigan cardiologists argue that this shift needs a different way of thinking about risk.
The authors, Eric D. Smith, MD, and Venkatesh L. Murthy, MD, PhD, both study gene therapy at the University of Michigan Health Frankel Cardiovascular Center. Their paper, "Clinical Framework for Durable Genomic Interventions," proposes five questions to ask before a one-time genetic treatment is used widely.
Why rare diseases are the easier case
For a rare, severe genetic disease, the choice is often simple. Patients may have no other treatment, and the disease itself is dangerous, so a one-time therapy with some unknown long-term risk can still be the best option. There are more than 6,000 known rare genetic disorders, although only a fraction have a target that current gene therapies can reach.
Common conditions are different. People with high cholesterol, for example, already have effective drugs such as statins that can be adjusted or stopped. A permanent genetic treatment has to compete with those options, not with nothing.
Gene Tech Times has covered this trend, including a CRISPR infusion that kept cholesterol and triglycerides down for a full year and early data behind Lilly's bet on Verve.
Permanence can be a drawback
Many gene therapies and gene-editing treatments are designed to work for years, possibly for life, after a single dose. That is their main appeal. But the authors point out that permanence becomes a problem for conditions that need careful dose adjustment, or where the safe range is narrow.
A pill can be stopped if side effects appear. A permanent edit to DNA in liver cells cannot. "Until those safeguards are proven reliable, reversibility should be viewed as an important clinical benefit," Murthy said, referring to the "off switches" researchers are developing for genetic therapies.
Delivery adds another limit. Many therapies use viral vectors, and the immune response they trigger can prevent a second dose. (Read more in why gene therapy might not be a permanent cure.)
Small trials can miss rare side effects
Clinical trials of gene therapies are usually small and short. That is enough to show whether a treatment works, but not always enough to find side effects that are rare or appear years later.
"Clinical trials are often too small and too short to identify rare complications," Smith said. When a therapy is given to a few hundred people with a rare disease, a one-in-10,000 side effect may never appear. When it is given to millions of people with a common condition, that same side effect could harm hundreds.
The authors call for long-term, independent, standardized safety monitoring before genetic treatments are used broadly. Current therapies edit somatic cells, the body's non-reproductive cells, so the changes are not meant to pass to children. (See can gene therapy be passed on to future generations?)
Five questions to ask
The paper proposes five questions for judging whether a durable genetic treatment is a good fit for a disease:
How severe is the disease? The more serious the condition, the more risk is acceptable.
Are there reversible alternatives? If effective drugs that can be adjusted or stopped already exist, the bar is higher.
Is the target tissue suited to a one-time treatment? Some organs and cell types are better candidates than others.
Is the delivery method's risk proportionate? The risks of the vector or delivery system should match the benefit.
Can patients be monitored long-term? Durable treatments need durable follow-up.
The authors also raise cost. Some gene therapies list at more than $1 million per patient, and they argue that benefits should not come at the expense of equity. (Read more in why gene therapy is expensive.)
Not a call to slow down
The authors are clear that they support the field. "The goal is not to slow progress," Smith said. Their aim is a shared framework that doctors, regulators and patients can use as genetic treatments expand from rare diseases to conditions that affect millions of people.
The bottom line
One-time genetic treatments are moving from rare diseases to common ones such as high cholesterol. Two Michigan cardiologists argue that this calls for extra caution: weighing permanence against reversible drugs, watching for rare side effects over many years, and asking five questions about severity, alternatives, target tissue, delivery risk and monitoring before broad use.
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