Gene therapy for Parkinson's disease: now getting closer

BMC A11, Lund, Sweden Gene transfer in PD is currently pursued along two different lines: either by gene transfer of potential neuroprotective molecules, or as a tool to supply dopamine to the affected striatum by introducing the enzymes responsible for L-DOPA or dopamine synthesis. Promising results have been obtained in the rat 6-OHDA lesion model and the monkey MPTP lesion model by gene transfer using recombinant viral vectors, in particular the new generations of highly efficient adeno-associated virus (AAV) or lentiviral vectors carrying the GDNF or Tyrosine hydroxylase (TH) genes. Nanogram amounts of GDNF…