A single round of genome editing could protect a person for decades against cardiovascular disease, high cholesterol, obesity and diabetes, freeing them from a lifetime of pills. But an irreversible change to the genetic code carries delayed risks that may only surface years later, the website infohub.kz reports.
Cardiologists Erik D. Smith and Venkatesh L. Murthy of the Frankel Cardiovascular Center at the University of Michigan made the case in an article for the scientific journal JAMA Internal Medicine. As ScienceDaily notes, a one-time gene therapy could eventually replace daily medication for heart disease, but the scientists warn of hidden, delayed risks from permanent changes to DNA.
Unlike conventional pharmaceuticals, which work only while they are taken continuously, gene therapy alters DNA itself, introducing working copies of genes or blocking dangerous cellular activity. Such technologies have already proven highly effective against severe inherited disorders such as spinal muscular atrophy or sickle cell anemia, where a deadly threat justifies any side effects. Applying the approach to widespread diseases, however, poses serious challenges. A doctor can adjust the dose of an ordinary drug or stop it if a patient tolerates it poorly, whereas genetic modification of somatic cells cannot yet be quickly switched off.
The authors drew up a list of five mandatory criteria to meet before prescribing genomic therapy: an assessment of how severe the disease is, the availability of flexible alternatives, how long the effect lasts in tissues, the safety of vector delivery, and arrangements for multi-year monitoring. The researchers stressed that an immune response to viral vectors can block a repeat dose. Moreover, when millions of patients are treated, even the rarest complications could harm thousands of people, and the exorbitant cost of the therapy risks widening inequality in medicine.