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One-Dose Therapy Debate
Gene-editing therapies may make it possible to treat some diseases with a single, long-lasting dose instead of taking medication for life. They have made progress in rare diseases such as spinal muscular atrophy and sickle cell disease, while researchers are exploring their use for common conditions such as high cholesterol and diabetes. But the effects of genetic changes may be difficult to reverse, and immune complications or rare, delayed side effects may occur. Describing a treatment as “one-time” also does not guarantee that its effects will last a lifetime. Two researchers caution against expanding their use without long-term studies and strict safety standards, particularly given that some treatments cost more than a million dollars per patient.
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