Use of a histone deacetylase inhibitor for treating muscular...

A - Human Necessities – 61 – K

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A61K 31/19 (2006.01) A61K 31/16 (2006.01) A61K 31/192 (2006.01) A61K 31/198 (2006.01) A61K 31/202 (2006.01) A61K 31/223 (2006.01) A61K 31/325 (2006.01) A61K 31/473 (2006.01) A61K 38/12 (2006.01) A61P 21/00 (2006.01)

Patent

CA 2507450

The invention concerns the use of a histone deacetylase inhibitor for preparing a medicine for treating or preventing a disease resulting from deficiency of an adult gene in a subject by re-expression of a homologous fetal gene. The invention concerns particularly the treatment of dystrophies such as Duchenne's dystrophy or Becker's dystrophy where the defective gene is the dystrophin gene and the homologous fetal gene is the utrophin gene.

La présente invention concerne l'utilisation d'un inhibiteur d'histone déacétylase pour la préparation d'un médicament destiné au traitement ou à la prévention d'une maladie résultant de la déficience d'un gène adulte chez un individu par la ré-expression du gène foetal homologue. L'invention s'intéresse tout particulièrement au traitement des dystrophies comme la dystrophie de Duchenne ou la dystrophie de Becker où le gène adulte défectueux est le gène de la dystrophine et le gène foetal homologue est le gène de l'utrophine.

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