… splicing modification by antisense oligonucleotides, chaperone … Late-onset Pompe disease
(LOPD) is defined as GSDII … fact that glycogen accumulation in early-onset Pompe disease …

… reduced glycogen accumulation in patient-derived myotubes46. Other ASO studies have
sought to reduce glycogen by … Benefiting from advancements in ASO technology, here we …

… Wal et al. in Pompe patient-derived myotubes and fibroblasts, and an … reduction-based
therapy inhibiting glycogen synthesis could be used as an adjunctive strategy to ERT. Antisense …

… screen using the U7 snRNA expression system, while we used nucleofection to administer
the PMOs. In another study, Pompe patient-derived myotubes … of reduced glycogen levels 27 . …

… The use of antisense oligonucleotide (AON)-mediated … each other and form myotubes, or
to fuse with existing myotubes. … of alglucosidase alfa in late-onset Pompe’s disease. N. Engl. …

… , antisense oligonucleotides or inhibition of glycogen synthesis with substrate reduction therapy,
are … exon 2, as well as a decrease in glycogen in patient myotube cultures [42]. Similarly, …

… Pompe disease (glycogen storage disease type II) is caused … for patients with early and late
onset Pompe disease as well … in cultured GAA-deficient myotubes and in muscle of KO mice …

… Pompe disease, also known as glycogen storage disease … analysis in IPD patient fibroblasts,
myoblasts, and myotubes … treat either infantile-onset or late-onset Pompe disease patients. …

… Pompe like mouse myotubes were generated as model to verify the potency of a muscle homing
tag in uptake, lysosomal targeting, and glycogen reduction upon … gene therapy using the …

… Pompe disease (PD), or glycogen storage disease type II (GSD II), is an autosomal-… To
date, a dried blood spot (DBS) is used to detect GAA enzyme activity, and if it is reduced, genetic …