Use of a histone deacetylase inhibitor for treating muscular dystrophies
AssigneeCentre National de la Recherche Scientifique (CNRS)
InventorsSabine De La Porte, Maurice Israel, Vincent Voisin, Hafedh Haddad
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Abstract
The invention relates to an inhibiter of histone deacetylase for treating or preventing a disease resulting from the deficiency of an adult gene in an individual by the re-expression of the homologous fetal gene. The invention concerns in particular the treatment of dystrophies such as Duchenne's dystrophy or Becker's dystrophy in which the defective adult gene is the dystrophin gene and the homologous fetal gene is the utrophin gene.
Claims32
1-31. (canceled)
A method of treating or preventing a disease resulting from a deficiency of an adult gene for which there is a silent homologous gene comprising administering to a patient a therapeutically effective amount of an inhibitor of histone deacetylase that permits re-expression of the homologous gene.
A method of treating or preventing a disease resulting from a deficiency of an adult gene for which there is a silent homologous gene comprising administering to a patient a therapeutically effective amount of an inhibitor of histone deacetylase and a compound regulating a protein coded by the adult gene.
A product in which an inhibitor of histone deacetylase and a compound regulating a protein coded by an adult gene are connected by covalance, optionally with a spacer arm, which connection or spacer arm is cleavable in an organism in such a manner as to release two active ingredients.
Dependencies are inferred from the claim text.