Compositions and methods for the prevention and treatment of...

C - Chemistry – Metallurgy – 12 – Q

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C12Q 1/68 (2006.01) C07H 21/04 (2006.01) C12N 15/11 (2006.01) C12N 15/85 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2455424

The present invention provides compositions and methods for the prevention and treatment of a neurodegenerative disease, specifically Huntington's disease. In particular, the invention provides single-stranded, modified oligonucleotides for the targeted alteration of the genetic sequence of the Huntington's disease gene, and methods of treating or preventing Huntington's disease using the same.

L'invention porte sur des compositions et procédés de prévention et traitement de maladies neurodégénératives, et spécialement de la maladie de Huntington à cette fin l'invention utilise des oligonucléotides monobrin modifiés en vue d'une altération ciblée de la séquence génétique du gène de la maladie de Huntington, et sur des procédés de traitement et prévention de ladite maladie les utilisant.

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