CHIMERIC PROMOTER FOR CONTROLLING EXPRESSION IN SKELETAL MUSCLE CELL

PROBLEM TO BE SOLVED: To provide a gene construct, etc., useful for gene therapy for treating muscle-affecting diseases, including cardiovascular disorders. SOLUTION: This present invention concerns chimeric constructs comprising a skeletal α-actin gene promoter operably linked with at least a skele...

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Hauptverfasser: RIBAULT SEBASTIEN, NEUVILLE PASCAL, CALENDA VALERIE, FRAULI MELANIE
Format: Patent
Sprache:eng
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Zusammenfassung:PROBLEM TO BE SOLVED: To provide a gene construct, etc., useful for gene therapy for treating muscle-affecting diseases, including cardiovascular disorders. SOLUTION: This present invention concerns chimeric constructs comprising a skeletal α-actin gene promoter operably linked with at least a skeletal muscle- specific enhancer of a human gene. It also provides an expression cassette comprising such a chimeric construct to control expression of a gene of interest. The invention also relates to a vector, a viral particle, an eukaryotic host cell, a pharmaceutical composition each comprising the expression cassette and their use for specific expression in skeletal muscle cells as well as for therapeutic or prophylactic purposes. Finally, the present invention also provides the therapeutic use of an expression cassette, a vector and a viral particle comprising a gene of interest placed under the control of a skeletal α-actin gene promoter and a muscle-specific enhancer, especially for treating peripheral ischemia. COPYRIGHT: (C)2003,JPO