IN VIVO GENE THERAPY OF PARKINSON'S DISEASE

摘要

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.