Gene therapy in Parkinson’s disease

References

• Hacein-Bey-Abina S, Le Deist F, Carlier F eta! Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl. I Merl 346(16), 1185–1193 (2002).
   Google Scholar
• Kaiser J. Gene therapy. Seeking the cause of induced leukemias in X-SCID trial. Science 299, 495 (2003).
   Google Scholar
• Leone P, Janson CG, Bilaniuk L et al. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease. Ann. Neural. 48, 27–38 (2000).
   PubMed Web of Science ®Google Scholar
• During MJ, Naegele JR, O'Malley KL, Geller AT. Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266, 1399–403 (1994).
   Google Scholar
• Horenou P, Vigne E, Castel MN et al Direct intracerebral gene transfer of an adenoviral vector expressing tyrosine hydroxylase in a rat model of Parkinson's disease. Neuroreport 6, 49–53 (1994).
   PubMed Web of Science ®Google Scholar
• Kaplitt MG, Leone P, Samulski RJ et al Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genet. 8,148–154 (1994).
   PubMed Web of Science ®Google Scholar
• Horellou P, Brundin P, Kalen P, Mallet J, Bjorklund A. hi vivo release of dopa and dopamine from genetically engineered cells grafted to the denervated rat striatum. Neuron 5,393–402 (1990).
   Google Scholar
• Fisher LJ, Jinnah HA, Kale LC, Higgins GA, Gage FH. Survival and function of intrastriatally grafted primary fibroblasts genetically modified to produce L-dopa. Neuron 6,371–380 (1991).
   Google Scholar
• Kink D, Georgievska B, Burger C eta]. Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer. Proc. Nat! Acad. Scl USA 99,4708–4713 (2002).
   Google Scholar
• Azzouz M, Martin-Rendon E, Barber RD eta! Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase and GTP cyclohydrolase I induces sustained transgene expression, dopamine production and functional improvement in a rat model of Parkinson's disease. j Neuroscl 22,10302–10312 (2002).
   PubMed Web of Science ®Google Scholar
• Lee WY, Chang JW, Nemeth NL, Kang UJ. Vesicular monoamine transporter-2 and aromatic L-amino acid decarboxylase enhance dopamine delivery after L-3,4-dihydroxyphenylalanine administration in Parkinsonian rats. j Neuroscl 19, 3266–3274 (1999).
   PubMed Web of Science ®Google Scholar
• During MJ, Samulski RJ, Elsworth JD et al hi viva expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Ther. 5,820–827 (1998).
   PubMed Web of Science ®Google Scholar
• Bankiewicz KS, Eberling JL, Kohutnicka M et al. Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using prodrug approach. Exp. Neural 164,2–14 (2000).
   PubMed Web of Science ®Google Scholar
• Freed CR, Greene PE, Breeze RE eta]. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N Engl. Merl 344,710–719 (2001).
   Google Scholar
• Olanow CW, Goetz CG, Kordower JH eta! A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann. Neural 54, 403–414 (2003).
   PubMed Web of Science ®Google Scholar
• Choi-Lundberg DL, Lin Q, Chang YN eta! Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science 275,838–841 (1997).
   PubMed Web of Science ®Google Scholar
• Kordower JH, Emborg ME, Bloch J et al Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 290, 767–773 (2000).
   PubMed Web of Science ®Google Scholar
• Nutt JG, Burchiel KJ, Comella CL eta! Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology60, 69–73 (2003).
   PubMed Web of Science ®Google Scholar
• Fitzsimons HL, Bland RJ, During MJ. Promoters and regulatory elements that improve adeno-associated virus transgene expression in the brain. Methods 28, 227–236 (2002).
   Google Scholar
• Gill SS, Patel NK, Hotton GR eta! Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson's disease. Nature Merl 9,589–595 (2003).
   PubMed Web of Science ®Google Scholar
• Luo J, Kaplitt MG, Fitzsimons HL eta! Subthalamic GAD gene therapy in a Parkinson's disease rat model. Science 298, 425–429 (2002).
   PubMed Web of Science ®Google Scholar
• Deep-brain stimulation for Parkinsonis Disease Study Group. Deep-brain stimulation of the subthalamic nucleus or the pars intema of the globus pallidus in Parkinsonis disease. N. Engl. Med. 345,956–963 (2001).
   PubMed Web of Science ®Google Scholar
• Umemura A, Jaggi JL, Hurtig HI et al Deep brain stimulation for movement disorders: morbidity and mortality in 109 patients. I Neurosurg. 98,779–784 (2003).
   PubMed Web of Science ®Google Scholar
• Levy R, Lang AE, Dostrovsky JO eta! Lidocaine and muscimol microinjections in subthalamic nucleus reverse Parkinsonian symptoms. Brain 124,2105–2118 (2001).
   PubMed Web of Science ®Google Scholar
• During MJ, Kaplitt MG, Stem MB,Eidelberg D. Subthalamic GAD gene transfer in Parkinson's disease patients who are candidates for deep brain stimulation. Hum. Gene Ther 12,1589–1591 (2001).
   Google Scholar
• Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Nat! Acad. Sri. USA 99,11854–11859 (2002).
   PubMed Web of Science ®Google Scholar
• Watson DJ, Kobinger GP, Passini MA, Wilson JM, Wolfe JH. Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins. Mal Ther. 5,528–537 (2002).
   Google Scholar
• Trojanowski JQ, Lee VM. Parkinson's disease and related a-synucleinopathies are brain amyloidoses. Ann. NY Acad. Sri. 991, 107–110 (2003).Website
   Google Scholar
• http://news.independent.co.ulduldhealth/ storyjsp?story = 451061 (Accessed October 2003)
   Google Scholar