Methods and compositions for use in gene therapy for...

A - Human Necessities – 61 – K

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A61K 48/00 (2006.01) A61K 38/48 (2006.01) C12N 7/01 (2006.01) C12N 9/64 (2006.01) C12N 15/63 (2006.01) C12N 15/86 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2283639

The invention includes a composition comprising a recombinant adeno-associated viral vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding Factor IX and accompanying 5' and 3' untranslated regions and a transcription termination signal, and methods of use thereof.

L'invention concerne une composition comprenant un vecteur viral recombiné adéno-associé présentant au moins deux répétitions terminales inversées de virus adéno-associé, une séquence promoteur/régulatrice, de l'ADN isolé codant le facteur IX et accompagnant les régions 5' et 3' non traduites ainsi qu'un signal d'achèvement de transcription, et ses modes d'utilisation.

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