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Gene therapy is no longer the future of medicine—it's reality. Treatments that replace, silence or edit genes are already changing clinical care.

There are more than 6,000 rare genetic disorders, but only a fraction currently have treatable targets.

Early successes in gene therapy have focused on these rare disorders, such as spinal muscular atrophy and sickle cell disease. But research is rapidly moving toward genomic therapies to treat common conditions, including high cholesterol, heart disease, obesity and diabetes.

When is gene therapy the right choice? When does the potential benefit of gene editing outweigh possible permanent harm?

In a recent 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., proposed strategies for determining when genomic therapy is justified.

Here, the two cardiologists, who study gene therapy, answer questions about the promise surrounding these treatments and the risks that remain as gene editing moves into the mainstream.

Smith: Conventional medications work only while patients continue taking them. Genomic therapies can supply a functional copy of a gene, reduce the activity of a harmful gene or directly edit a DNA sequence.

Because they act at the level of genetic material, many are designed as single-administration, or "one-and-done," treatments. But that does not guarantee their benefits will last forever. Many of these therapies rely on viral delivery vehicles, and the body's immune response often prevents a second dose from being given even if one were needed.

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