Use of apoptosis inhibiting compounds in degenerative...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/85 (2006.01) A61K 31/7088 (2006.01) A61K 31/713 (2006.01) A61K 38/17 (2006.01) A61K 48/00 (2006.01) A61P 25/28 (2006.01) C07K 14/47 (2006.01) C07K 19/00 (2006.01) C12N 15/12 (2006.01) C12N 15/86 (2006.01)

Patent

CA 2584986

The invention provides methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain associated with a neurodegenerative diseases that is characterized by an excess buildup of intracellular protein aggregates. In particular, the invention provides methods and compositions used to deliver an adeno- associated virus vector (AAV) comprising a nucleotide sequence encoding an inhibitor of apoptosis protein (IAP) to cells in the region.

L'invention concerne des méthodes et des compositions permettant l'administration localisée d'un vecteur contenant un agent thérapeutique à une zone spécifique du cerveau, associée aux maladies neurodégénératives, et qui se caractérise par une accumulation excessive d'agrégats de protéines intracellulaires. L'invention concerne en particulier des méthodes et des compositions destinées à administrer à des cellules dans cette zone du cerveau un vecteur de virus associé aux adénovirus (VAA) contenant une séquence nucléotidique codant un inhibiteur de la protéine de l'apoptose (IAP).

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