A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 48/00 (2006.01) A61K 38/37 (2006.01) C07H 21/04 (2006.01) C12N 15/00 (2006.01) C12N 15/63 (2006.01) C12N 15/864 (2006.01)
Patent
CA 2348068
The present invention provides improved viral vectors useful for the expression of genes at high levels in human cells. In particular, the present invention provides adeno-associated vectors (AAV) suitable for gene therapy. These vectors are capable of delivering nucleic acid containing constructs which result in the production of full-length therapeutic levels of biologically active Factor VIII in the recipient individual in vivo. The present invention also provides pharmaceutical compositions comprising such AAV vectors, as well as methods for making and using these constructs.
L'invention concerne des vecteurs viraux améliorés, utiles pour l'expression de gènes, à des niveaux élevés, dans des cellules humaines. L'invention concerne notamment des vecteurs adéno-associés (AAV) se prêtant à une thérapie génique. Ces vecteurs sont capables de délivrer des constructions contenant de l'acide nucléique, ce qui donne lieu à la production, à des niveaux thérapeutiques intégrales, du facteur VIII biologiquement actif, chez le sujet receveur in vivo. L'invention concerne en outre des compositions pharmaceutiques contenant lesdits vecteurs AAV, ainsi que des méthodes de production et d'utilisation desdites constructions.
Colosi Peter C.
Couto Linda B.
Qian Xiaobing
Avigen Inc.
Smart & Biggar
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