Design of new nano-dysferlin molecules for AAV therapy
Correcting point mutations in the DYSF gene with Prime editing
Galectin-1: A potential protein therapy for Limb Girdle Muscular Dystrophy type 2B
Determining efficacy of 4-phenylbutyric acid in in vitro and in vivo models of dysferlinopathy
Cutting the fat: Targeting DGAT2 to treat dysferlin-deficient muscle
Analyzing the role of dysferlin in skeletal muscle in membrane repair
A Concept Elicitation/ Feasibility Study for the Limb-Girdle Video Assessment
Lipidomic analyses of dysferlin-deficient muscles related to age, myofibre type, sex
Biochemical and Therapeutic Characterization of Dysferlin
Monocyte assay and targeted transcriptome sequencing as a functional tool for dysferlin detection and dysferlinopathy molecular diagnosis