Means and methods for the specific modulation of target...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/11 (2006.01) A01K 67/027 (2006.01) A61K 9/08 (2006.01) A61K 31/713 (2006.01) A61K 38/17 (2006.01) A61K 48/00 (2006.01) C07K 14/47 (2006.01) C07K 16/18 (2006.01) C12N 15/12 (2006.01) C12Q 1/68 (2006.01) G01N 33/50 (2006.01) G01N 33/68 (2006.01)

Patent

CA 2482904

Provided are methods for the treatment of disorders of the central nervous system (CNS) and the eye. In particular, use of compositions comprising a compound capable of modulating a target gene or gene product is described for the preparation of a pharmaceutical composition for the treatment of disorders of the CNS and/or the eye, wherein the composition is designed to be administered outside the blood-CNS and the blood-retina barriers. Furthermore, methods are provided for identifying and obtaining nucleic acid molecules encoding polypeptides involved in CNS disorders or of the eye, methods for diagnosing said disorders as well as transgenic animal deficient in the expression of target genes identified in accordance with the described method. In addition, methods of identifying and isolating drugs that are particularly useful for the treatment of disorders related to the CNS and/or the eye are disclosed.

L'invention concerne des procédés pour le traitement de troubles du système nerveux central (SNC) et de l'oeil. Elle concerne en particulier l'utilisation de compositions, contenant un composé pouvant moduler un gène ou un produit génique cible, pour la préparation d'une composition pharmaceutique destinée au traitement de troubles du système nerveux central (SNC) et/ou de l'oeil, ladite composition pharmaceutique étant conçue pour être administrée à l'extérieur des barrières hémato-SNC et hémato-rétiniennes. L'invention concerne en outre des procédés pour identifier et obtenir des molécules d'acides nucléiques codant des polypeptides impliqués dans les troubles du SNC ou de l'oeil, des procédés pour diagnostiquer lesdits troubles, ainsi qu'un animal transgénique présentant une déficience d'expression des gènes cibles identifiés selon les procédés mentionnés. L'invention concerne également des procédés pour identifier et isoler des médicaments particulièrement utiles pour le traitement de troubles du système nerveux central (SNC) et/ou de l'oeil.

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