Utrophin gene expression

Details Utrophin gene expression Utrophin gene expression

C - Chemistry, Metallurgy

12

N

C12N 15/12 (2006.01) A01K 67/027 (2006.01) A61K 35/34 (2006.01) A61K 38/17 (2006.01) A61K 48/00 (2006.01) C07K 14/47 (2006.01) C12N 5/10 (2006.01)

Patent

CA 2240938

Nucleic acid from which a polypeptide with utrophin function can be expressed, especially mini-genes and chimaeric constructs. Expression significantly decreases the severity of the dystrophic muscle phenotype in an animal model, indicating usefulness in treatment of muscular dystrophy. The nucleic acid and encoded polypeptides are also useful in screening for substances to modulate utrophin binding to actin and/or the dystrophin protein complex.

Acide nucléique à partir duquel peut être exprimé un polypeptide à fonction d'utrophine, en particulier des mini-gènes et des produits de recombinaison chimériques. Ladite expression réduit de manière importante la gravité du phénotype de muscle dystrophique chez un modèle animal, ce qui constitue une indication de son utilité dans le traitement de la dystrophie musculaire. Ledit acide nucléique et les polypeptides codés sont également utiles pour cribler des substances destinées à moduler la liaison de l'utrophine à l'actine et/ou au complexe protéinique de la dystrophine.

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