Genomic medicine corrects common LOPD defect in cells, mice

Genomic medicine corrects common LOPD defect in cells, mice

A genomic medicine designed to correct the genetic defect found in up to 90% of people with late-onset Pompe disease (LOPD) worked as intended in patient-derived muscle cells and a mouse model, according to a study. The study, “Splicing correction by peptide-conjugated morpholinos as a novel treatment for Late-Onset Pompe Disease,” was published in Molecular Therapy Nucleic Acids. It was funded by Sarepta Therapeutics, and all study authors are …