FIELD: biotechnology.
SUBSTANCE: disclosed is an antibody or a binding fragment thereof, which specifically bind to a glucose carrier protein 4 (GLUT4). Invention also relates to compositions and methods for delivering a payload into cells or tissues which express GLUT4. Compositions contain said antibody or its binding fragment conjugated with a therapeutic payload. Payload contains a nucleic acid which codes a functional dystrophin protein or a functional fragment thereof. Compositions according to the invention are applicable for methods of treating a disease or condition in a subject with a genetic mutation in a gene coding a dystrophin protein.
EFFECT: invention provides non-viral vector platforms which can specifically deliver a therapeutic payload to muscle cells without payload size limitations.
27 cl, 25 dwg, 3 tbl
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Authors
Dates
2025-01-21—Published
2020-07-09—Filed