title: [Studies of gene therapy for Parkinson's disease].
abstract: Currently, four Phase I clinical trials are underway utilizing recombinant adeno-associated viral (rAAV) vectors for the treatment of Parkinson's disease. The vehicle used mainly for gene delivery to the human brain is rAAV vector, which is non-pathogenic and non-self-amplifying. At present, the gene therapy approach is not the best way for the treatment of PD patients, but we believe that the further progress is anticipated toward making this strategy a therapeutic option for PD in the future. This article will review currently ongoing clinical trials of PD gene therapy and then introduce our studies about the gene therapy for PD.
authors.affiliation: Department of Neurology, Kitasato University.
authors.forename: Hideki
authors.initials: H
authors.lastname: Mochizuki
mesh_terms: D000818:Animals; D017321:Clinical Trials, Phase I as Topic; D000229:Dependovirus; D004195:Disease Models, Animal; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000639:trends; D005822:Genetic Vectors; D006801:Humans; D008928:Mitochondria; D009154:Mutation; D010300:Parkinson Disease / Q000628:therapy*; D044968:Regenerative Medicine / Q000639:trends; D044767:Ubiquitin-Protein Ligases / Q000235:genetics
chemical_list: D044767:Ubiquitin-Protein Ligases; C111567:parkin protein