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Innate immune responses to systemically administered adeno-associated virus (AAV) gene therapy vectors have been implicated as potentiators of serious adverse events, and are a significant […]
Background: Delandistrogene moxeparvovec is an rAAVrh74 vector-based gene transfer therapy that delivers a transgene encoding delandistrogene moxeparvovec micro-dystrophin, an engineered, functional form of dystrophin shown […]
Background: Delandistrogene moxeparvovec (rAAVrh74 vector-based gene therapy approved in the US and other select countries) delivers a transgene encoding micro-dystrophin, an engineered, functional form of […]
The Russian COL-6 register was created in January 2024 by Russian COL-6 patient community . On the day of its creation, it included 74 patients with COL-6. […]
Hypothesis: Modifying the acid-α-glucosidase (GAA) gene at histidine 201 (H201) with hydrophobic (isoleucine, leucine) and amphipathic (tyrosine, serine) residues would improve lysosomal processing and increase […]
GNT0004 is an AAV-8 based gene therapy, containing a shortened functional dystrophin gene (hMD1) with a Spc5.12 promoter, targeting skeletal and cardiac muscles for treating […]
Background: CD93, a type 1 transmembrane glycoprotein with pro-inflammatory properties, is highly expressed in macrophages and endothelial cells during advanced inflammation. The pathogenesis of Duchenne […]
Introduction Congenital muscle diseases have a diverse array of clinical, genetic and histopathologic features (1). Although past cohorts have described features of these disease (2), […]
Oculopharyngeal Muscular Dystrophy (OPMD) is a rare, autosomal dominant, late-onset degenerative muscle disorder presenting in patients at 40-60 years of age. OPMD is principally characterized […]