Substrate-reducing ASOs show promise in Pompe mouse model

A new type of substrate reduction therapy composed of antisense oligonucleotides, or ASOs, effectively reduces glycogen accumulation in the skeletal muscles, or those attached to bones, of a Pompe disease mouse model, a study found. When combined with standard enzyme replacement therapy (ERT), which showed limited…

Molecular ‘Tag’ Improves Efficacy of Gene Therapy for Pompe Disease in Mice, Study Shows

A molecular “tag” to improve distribution of the acid-alpha glucosidase (GAA) protein within cells enhanced the efficacy of gene therapy in a mouse model of Pompe disease, a recent study suggests. The study, titled “AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the…