Current status of gene therapy for parkinson disease

Brain and Nerve

Volumn 61, Issue 4, 2009, Pages 485-493

  Mochizuki, Hideki ^a  

^a JUNTENDO UNIVERSITY   (Japan)

Author keywords

AAV; Gene therapy; Parkin; Parkinson disease

Indexed keywords

ADENOVIRUS VECTOR; LENTIVIRUS VECTOR; PARKIN; UBIQUITIN PROTEIN LIGASE E3;

ARTICLE; CLINICAL EXAMINATION; CLINICAL TRIAL; DOPAMINERGIC NERVE CELL; DRUG DELIVERY SYSTEM; DRUG EFFICACY; GENE THERAPY; GENE TRANSFER; HUMAN; NONHUMAN; PARKINSON DISEASE;

ANIMALS; APOPTOTIC PROTEASE-ACTIVATING FACTOR 1; CLINICAL TRIALS AS TOPIC; DEPENDOVIRUS; GENE THERAPY; GENETIC VECTORS; GLUTAMATE DECARBOXYLASE; HUMANS; NEURTURIN; PARKINSON DISEASE; RATS; UBIQUITIN-PROTEIN LIGASES;

EID: 64849110996     PISSN: 18816096     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (41)

1. ) Kaneda N, Kobayashi K, Ichinose H, Kishi F, Nakazawa A, et al: Isolation of a novel cDNA clone for human tyrosine hydroxylase: alternative RNA splicing produces four kinds of mRNA from a single gene. Biochem Biophys Res Commun 146: 971-975, 1987
2. ) Uchida K, Takamatsu K, Kaneda N, Tova S, Tsukada Y, et al: Synthesis of L-3, 4-dihydroxyphenylalanine by tyrosine hydroxylase cDNA-transfected C6 cells: application for intracerebral grafting. J Neurochem 53: 728-732, 1989
3. Wolff JA, Fisher LJ, Xu L, Jinnah HA, Langlais PJ, et al: Grafting fibroblasts genetically modified to produce L-dopa in a rat model of Parkinson disease. Proc Natl Acad Sci U S A 86: 9011-9014, 1989
4. Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, et al: Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 8: 148-154, 1994
5. During MJ, Naegele JR, O'Malley KL, Geller AI: Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266: 1399-1403, 1994
6. Akli S, Caillaud C, Vigne E, Stratford-Perricaudet LD, Poenaru L, et al: Transfer of a foreign gene into the brain using adenovirus vectors. Nat Genet 3: 224-228, 1993
7. Horellou P, Vigne E, Castel MN, Barnéoud P, Colin P, 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
8. Naldini L, Blomer U, Gallay P, Orv D, Mulligan R, et al: In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272: 263-267, 1996
9. Mochizuki H, Schwartz JP, Tanaka K, Brady RO, Reiser J: High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J Virol 72: 8873-8883, 1998
10. Kordower JH, Emborg ME, Bloch J, Ma SY, Chu Y, et al: Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 290: 767-773, 2000
11. Yamada M, Onodera M, Mizuno Y, Mochizuki H: Neurogenesis in olfactory bulb identified by retroviral labeling in normal and l-methyl-4-phenyl-l, 2, 3, 6- tetrahydropyridine-treated adult mice. Neuroscience 124: 173-181, 2004
12. Suzuki A, Obi K, Urabe T, Hayakawa H, Yamada M, et al: Feasibility of ex vivo gene therapy for neurological disorders using the new retroviral vector GCDNsap packaged in the vesicular stomatitis virus G protein. J Neurochem 82: 953-960, 2002
13. Hayakawa H, Hayashita-Kinoh H, Nihira T, Seki T, Mizuno Y, et al: The isolation of neural stem cells from the olfactory bulb of Parkinson's disease model. Neurosci Res 57: 393-398, 2007
14. Takagi Y, Takahashi J, Saiki H, Morizane A, Haya- shi T, et al: Dopaminergic neurons generated from monkey embryonic stem cells function in a Parkinson primate model. J Clin Invest 115: 102-109, 2005
15. Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisa- ka T, et al: Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131: 861-872, 2007
16. Wernig M, Zhao JP, Pruszak J, Hedlund E, Fu D, et al: Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease. Proc Natl Acad Sci U S A 105: 5856-5861, 2008
17. Haber B, Sze PY, Kuriyama K, Roberts E: GABA as a repressor of L-glutamic acid decarboxylase (GAD) in developing chick embryo optic lobes. Brain Res 18: 545-547, 1970
18. Luo J, Kaplitt MG, Fitzsimons HL, Zuzga DS, Liu Y, et al: Subthalamic GAD gene therapy in a Parkinson's disease rat model. Science 298: 425-429, 2002
19. Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, et al: Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369: 2097-2105, 2007
20. Feigin A, Kaplitt MG, Tang C, Lin T, Mattis P, et al: Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease. Proc Natl Acad Sci U S A 104: 19559-19564, 2007
21. Kang UJ, Fisher LJ, Joh TH, O'Malley KL, Gage FH: Regulation of dopamine production by genetically modified primary fibroblasts. J Neurosci 13: 5203- 5211, 1993
22. During MJ, Samulski RJ, Elsworth JD, Kaplitt MG, Leone P, et al: In vivo 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
23. Eberling JL, Jagust WJ, Christine CW, Starr P, Larson P, et al: Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 70: 1980-1983, 2008
24. Azzouz M, Martin-Rendon E, Barber RD, Mitrophanous KA, Carter EE, et al: 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 Neurosci 22: 10302-10312, 2002
25. Bohn MC: A commentary on glial cell line-derived neurotrophic factor (GDNF). From a glial secreted molecule to gene therapy. Biochem Pharmacol 57: 135-142, 1999
26. Gasmi M, Herzog CD, Brandon EP, Cunningham JJ, Ramirez GA, et al: Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's disease. Mol Ther 15: 62-68, 2007
27. Marks WJ Jr, Ostrem JL, Verhagen L, Starr PA, Larson PS, et al: Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol 7: 400-408, 2008
28. Mizuno Y, Ikebe S, Hattori N, Nakagawa-Hattori Y, Mochizuki H, et al: Role of mitochondria in the etiology and pathogenesis of Parkinson's disease. Biochim Biophys Acta 1271: 265-274, 1995
29. Kanuka H, Sawamoto K, Inohara N, Matsuno K, Okano H, et al: Control of the cell death pathway by Dapaf-1, a Drosophila Apaf-l/CED-4-related caspase activator. Mol Cell 4: 757-769, 1999
30. Mochizuki H, Hayakawa H, Migita M, Shibata M, Tanaka R, et al: An AAV-derived Apaf-1 dominant negative inhibitor prevents MPTP toxicity as antiapoptotic gene therapy for Parkinson's disease. Proc Natl Acad Sci U S A 98: 10918-10923, 2001
31. Kitada T, Asakawa S, Hattori N, Matsumine H, Yamamura Y, et al: Mutations in the parkin gene cause autosomal recessive juvenile parkinsonism. Nature 392: 605-608, 1998
32. Shimura H, Hattori N, Kubo S, Mizuno Y, Asakawa S, et al: Familial Parkinson disease gene product, parkin, is a ubiquitin-protein ligase. Nat Genet 25: 302-305, 2000
33. Petrucelli L, O'Farrell C, Lockhart PJ, Baptista M, Kehoe K, et al: Parkin protects against the toxicity associated with mutant alpha-synuclein: proteasome dysfunction selectively affects catecholaminergic neurons. Neuron 36: 1007-1019, 2002
34. Yamada M, Iwatsubo T, Mizuno Y, Mochizuki H: Overexpression of alpha-synuclein in rat substantia nigra results in loss of dopaminergic neurons, phosphorylation of alpha-synuclein and activation of caspase-9: resemblance to pathogenetic changes in Parkinson's disease. J Neurochem 91: 451-461, 2004
35. Yamada M, Mizuno Y, Mochizuki H: Parkin gene therapy for alpha-synucleinopathy: a rat model of Parkinson's disease. Hum Gene Ther 16: 262-270, 2005
36. Mochizuki H: Gene therapy for Parkinson's disease. Expert Rev Neurother 7: 957-960, 2007
37. Lo Bianco C, Schneider BL, Bauer M, Sajadi A, Brice A, et al: Lentiviral vector delivery of parkin prevents dopaminergic degeneration in an alpha-synuclein rat model of Parkinson's disease. Proc Natl Acad Sci U S A 101: 17510-17515, 2004
38. Vercammen L, Van der Perren A, Vaudano E, Gijsbers R, Debvser Z, et al: Parkin protects against neurotoxicity in the 6-hydroxydopamine rat model for Parkinson's disease. Mol Ther 14: 716-723, 2006
39. Winklhofer KF: The parkin protein as a therapeutic target in Parkinson's disease. Expert Opin Ther Targets 11: 1543-1552, 2007
40. Butcher J: Parkin gene therapy could treat Parkinson's disease. Lancet Neurol 4: 82, 2005
41. Yasuda T, Miyachi S, Kitagawa R, Wada K, Nihira T, et al: Neuronal specificity of alpha-synuclein toxicity and effect of parkin co-expression in primates. Neuroscience 144: 743-753, 2007