Application | Comment | Organism |
---|---|---|
medicine | Sleeping Beauty (SB) transposon is a nonviral gene transfer vector, already used in clinical trials. Full-length dysferlin transfer by the hyperactive sleeping beauty transposase restores dysferlin-deficient muscle, which can be used for nonviral gene delivery of full-length human dysferlin into muscle cells, along with a successful and efficient transplantation into skeletal muscle, important advances towards successful gene therapy of dysferlin-deficient muscular dystrophy | Homo sapiens |
Natural Substrates | Organism | Comment (Nat. Sub.) | Natural Products | Comment (Nat. Pro.) | Rev. | Reac. |
---|---|---|---|---|---|---|
additional information | Homo sapiens | H2K A/J muscle cells properly express full-length human DYSF following SB-mediated gene transfer. A duplicate of Spc5-12 (2xSpc5-12) regulatory sequence proves to be the most efficient in driving transgene expression in H2K A/J myoblasts. Corrected H2K A/J myoblasts can efficiently be transplanted into Scid/BLA/J mouse muscle | ? | - |
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Organism | UniProt | Comment | Textmining |
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Homo sapiens | - |
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- |
Substrates | Comment Substrates | Organism | Products | Comment (Products) | Rev. | Reac. |
---|---|---|---|---|---|---|
additional information | H2K A/J muscle cells properly express full-length human DYSF following SB-mediated gene transfer. A duplicate of Spc5-12 (2xSpc5-12) regulatory sequence proves to be the most efficient in driving transgene expression in H2K A/J myoblasts. Corrected H2K A/J myoblasts can efficiently be transplanted into Scid/BLA/J mouse muscle | Homo sapiens | ? | - |
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Synonyms | Comment | Organism |
---|---|---|
sleeping beauty transposase | - |
Homo sapiens |
General Information | Comment | Organism |
---|---|---|
physiological function | Sleeping Beauty (SB) transposon is a nonviral gene transfer vector, already used in clinical trials. Full-length dysferlin transfer by the hyperactive sleeping beauty transposase restores dysferlin-deficient muscle, which can be used for nonviral gene delivery of full-length human dysferlin into muscle cells, along with a successful and efficient transplantation into into skeletal muscle to cure dysferlin-deficient muscular dystrophy by gene therapy. Dysferlin-deficient muscular dystrophy is a progressive disease characterized by muscle weakness and wasting caused by mutations in DYSF, a large, multiexonic gene that forms a coding sequence of 6.2 kb. The hyperactive SB system consists of a transposon DNA sequence and a transposase protein, SB100X, that can integrate DNA over 10 kb into the target genome | Homo sapiens |