Ribozymal nucleic acids cleaving ccr5 or cxcr4

C - Chemistry – Metallurgy – 12 – N

Patent

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C12N 15/11 (2006.01) A61K 31/70 (2006.01) C07H 21/04 (2006.01) C12N 9/00 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2317895

A vector system which can be incorporated in liposomes, comprising at least one DNA vector, the vector or vectors containing a target-cleaving hammerhead ribozymal DNA sequence under control of a promoter effective in human cells and which, upon transcription to RNA will cleave the mRNA transcribed from a target gene encoding the CCR5 or CXCR4 protein. Preferably the ribozymal DNA contains a first recognition sequence (5' to 3'): tagattg or ctcact, respectively for CCR5 or CXCR4 and downstream thereof a second recognition sequence acttg or acgttgt respectively for CCR5 or CXCR4.

Système vecteur pouvant être incorporé aux liposomes, qui comprend au moins un vecteur d'ADN, le ou les vecteurs renfermant une séquence d'ADN ribozymal en marteau de clivage de site, sous le contrôle d'un promoteur actif dans les cellules humaines et qui, après transcription en ARN, clive l'ARNm transcrit à partir d'un gène cible codant pour la protéine CCR5 ou CXCR4. De préférence, l'ADN ribozymal contient une première séquence de reconnaissance (5' vers 3') tagattg ou ctcact, pour CCR5 ou CXCR4 respectivement, et, en aval de ladite séquence, une seconde séquence de reconnaissance acttg ou acgttgt, pour CCR5 ou CXCR4 respectivement.

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