Mtmr2-s polypeptide for use in the treatment of myopathies
AssigneesUniversite De Strasbourg, Centre National De La Recherche Scientifique, Institut National de la Sante et de la Recherche Medicale
InventorsJOCELYN LAPORTE, BELINDA COWLING, MATTHIEU RAESS, SYLVIE FRIANT-MICHEL, DIMITRI BERTAZZI
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Abstract
The present disclosure relates to a MTMR2-S polypeptide, or a nucleic acid sequence producing or encoding said MTMR2-S polypeptide, for a use in the treatment of a disease or disorder associated with MTM1 mutation or deficiency. The present invention provides compositions and methods for treatment of myopathy or diseases or disorders associated with MTM1 mutation or deficiency, in a subject in need thereof. The present invention relates to a method of delivering the MTMR2-S polypeptide to subjects in need of improved muscle function, such as subjects with centronuclear myopathies.
Claims21
1-12. (canceled)
A method for treating a disease or disorder associated with MTM1 mutation or deficiency in a subject in need thereof, said method comprising the step of administering to said subject a therapeutically effective amount of a MTMR2-S polypeptide or of a nucleic acid sequence producing or encoding said MTMR2-S polypeptide.
A pharmaceutical composition comprising a MTMR2-S polypeptide or a nucleic acid sequence producing or encoding said MTMR2-S polypeptide.
A nucleic acid construct, recombinant expression vector, or recombinant host cell comprising a nucleic acid sequence producing or encoding a MTMR2-S polypeptide;
- operably linked to one or more control sequences that direct the production of the said polypeptide.
Dependencies are inferred from the claim text.