A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 48/00 (2006.01) A61P 25/16 (2006.01) C07K 14/475 (2006.01) C12N 5/18 (2006.01) C12N 15/12 (2006.01) C12N 15/867 (2006.01)
Patent
CA 2543189
The present invention concerns methods and compositions for gene therapy, in particular in vivo gene therapy for delivery of bioactive Neurturin for the treatment of Parkinson's Disease. In another aspect the invention relates to virus expression constructs comprising a mammalian si( nal peptide linked to a mature or N-terminally truncated Neurturin without a functional proregion between the signal peptide and the Neurturin. These viral expression constructs are required for efficient secretion of bioactive Neurturin in in vivo gene therapy. The invention also concerns mammalian cells capable of producing Neurturin in increased amounts as well as the use of these cells for recombinant production of bioactive Neurturin and for therapeutic use.
L'invention concerne des procédés et des compositions pour la thérapie génique, en particulier la thérapie génique in vivo pour la délivrance de neurturine bioactive dans le traitement de la maladie de Parkinson. Selon un autre aspect, l'invention concerne des constructions d'expression virale qui comportent un peptide de signal mammalien lié à de la neurturine mature ou tronquée en terminaison N sans prorégion fonctionnelle entre le peptide et la neurturine. Ces constructions sont requises pour la sécrétion efficace de neurturine bioactive dans la thérapie génique in vivo. L'invention concerne également des cellules mammaliennes capables de produire de la neurturine en quantités accrues et elle concerne l'utilisation de ces cellules pour une production de recombinaison de neurturine bioactive et pour un usage thérapeutique.
Rosenblad Carl
Tornoe Jens
Wahlberg Lars
Gowling Lafleur Henderson Llp
Nsgene A/s
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