biostudies-literatureDalla Barba FUniversity of Ferrara research grantsAssociation Françoise contre les MyopathiesMuscular Dystrophy Association12707https://www.ebi.ac.uk/biostudies/studies/S-EPMC10454440biostudies-literatureUnknown24(16)Sarcoglycanopathies, also known as limb girdle muscular dystrophy 3-6, are rare muscular dystrophies characterized, although heterogeneous, by high disability, with patients often wheelchair-bound by late adolescence and frequently developing respiratory and cardiac problems. These diseases are currently incurable, emphasizing the importance of effective treatment strategies and the necessity of animal models for drug screening and therapeutic verification. Using the CRISPR/Cas9 genome editing technique, we generated and characterized δ-sarcoglycan and β-sarcoglycan knockout zebrafish lines, which presented a progressive disease phenotype that worsened from a mild larval stage to distinct myopathic features in adulthood. By subjecting the knockout larvae to a viscous swimming medium, we weInternational journal of molecular sciencesModeling Sarcoglycanopathy in <i>Danio rerio</i>.PMC1045444018620577888: doi.org/10.55762/pc.gr.8154023765FAR2022Scano MSandona DDalla Barba FLucon-Xiccato TArgenton FMouhib LSacchetto RSoardi MBertolucci CBenetollo ARichard IRisato GAkyurek EECarotti MfalseModeling Sarcoglycanopathy in <i>Danio rerio</i>.Sarcoglycanopathies, also known as limb girdle muscular dystrophy 3-6, are rare muscular dystrophies characterized, although heterogeneous, by high disability, with patients often wheelchair-bound by late adolescence and frequently developing respiratory and cardiac problems. These diseases are currently incurable, emphasizing the importance of effective treatment strategies and the necessity of animal models for drug screening and therapeutic verification. Using the CRISPR/Cas9 genome editing technique, we generated and characterized δ-sarcoglycan and β-sarcoglycan knockout zebrafish lines, which presented a progressive disease phenotype that worsened from a mild larval stage to distinct myopathic features in adulthood. By subjecting the knockout larvae to a viscous swimming medium, we we2023-01-01T00:00:00Z2023 Aug2025-07-10T03:08:15.648Z2025-04-04T07:13:43.771ZS-EPMC104544403762888810.3390/ijms241612707