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.
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.