Gene therapy shows promise in treating heart arrhythmia: Study

A pharmaceutical gene therapy has shown promise in treating calmodulinopathy, a rare and hard-to-treat heart arrhythmia, according to a study published Aug. 19 in Circulation

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Three genes — CALM1, CALM2 and CALM3 — are associated with heart function. A damaging variant in CALM1 causes calmodulinopathy. 

Researchers from Boston Children’s Hospital said they have found a “safe and effective” pharmaceutical therapy for treating the disease, according to a Sept. 18 news release from the hospital. The antisense oligonucleotide pharmaceutical treatment targets the CALM1 gene variant at the RNA level without affecting the other CALM genes. 

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