Gene therapy progress and prospects: Parkinson's disease

Gene Therapy

Volumn 10, Issue 20, 2003, Pages 1721-1727

  Burton, E A ^a   Glorioso, J C ^b   Fink, D J ^c,d  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b UNIVERSITY OF PITTSBURGH   (United States)

^c UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^d VETERANS AFFAIRS MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

4 AMINOBUTYRIC ACID; ADENOVIRUS VECTOR; ALPHA SYNUCLEIN; APOPTOSIS INHIBITOR; AROMATIC LEVO AMINO ACID DECARBOXYLASE; DOPAMINE; DOPAMINE RECEPTOR BLOCKING AGENT; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; GLUTAMATE DECARBOXYLASE; GUANOSINE TRIPHOSPHATE CYCLOHYDROLASE I; INHIBITOR OF APOPTOSIS PROTEIN; NEURONAL APOPTOSIS INHIBITORY PROTEIN; PARKIN; PROTEIN BCL 2; RECOMBINANTGLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; RETINOIC ACID RECEPTOR; TYROSINE 3 MONOOXYGENASE; UNCLASSIFIED DRUG; X LINKED INHIBITOR OF APOPTOSIS;

CLINICAL TRIAL; DNA MODIFICATION; DRUG EFFICACY; DRUG HALF LIFE; DRUG MECHANISM; DRUG SAFETY; DRUG TARGETING; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC CODE; HUMAN; NEUROPROTECTION; NONHUMAN; PARKINSON DISEASE; PRIORITY JOURNAL; REVIEW; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; APOPTOSIS; BRAIN; DOPAMINE; FORECASTING; GENE THERAPY; GENETIC VECTORS; GLIAL CELL LINE-DERIVED NEUROTROPHIC FACTOR; HUMANS; MODELS, ANIMAL; NERVE GROWTH FACTORS; PARKINSON DISEASE; STEM CELLS; TRANSDUCTION, GENETIC;

ADENOVIRIDAE;

EID: 0141516387     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302116     Document Type: Review

References (46)

1. Crocker SJ et al. NAIP protects the nigrostriatal dopamine pathway in an intrastriatal 6-OHDA rat model of Parkinson's disease. Eur J Neurosci 2001; 14: 391-400.
2. Mochizuki H 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 USA 2001; 98: 10918-10923.
3. Grondin R et al. Chronic, controlled GDNF infusion promotes structural and functional recovery in advanced parkinsonian monkeys. Brain 2002; 125: 2191-2201.
4. Date I et al. Grafting of encapsulated genetically modified cells secreting GDNF into the striatum of parkinsonian model rats. Cell Transplant 2001; 10: 397-401.
5. Akerud P, Canals JM, Snyder EY, Arenas E. Neuroprotection through delivery of glial cell line-derived neurotrophic factor by neural stem cells in a mouse model of Parkinson's disease. J Neurosci 2001; 21: 8108-8118.
6. Ostenfeld T et al. Neurospheres modified to produce glial cell line-derived neurotrophic factor increase the survival of transplanted dopamine neurons. J Neurosci Res 2002; 69: 955-965.
7. Park KW, Eglitis MA, Mouradian MM. Protection of nigral neurons by GDNF-engineered marrow cell transplantation. Neurosci Res 2001; 40: 315-323.
8. Shingo T, Date I, Yoshida H, Ohmoto T. Neuroprotective and restorative effects of intrastriatal grafting of encapsulated GDNF-producing cells in a rat model of Parkinson's disease. J Neurosci Res 2002; 69: 946-954.
9. Bensadoun JC et al. Lentiviral vectors as a gene delivery system in the mouse midbrain: cellular and behavioral improvements in a 6-OHDA model of Parkinson's disease using GDNF. Exp Neurol 2000; 164: 15-24.
10. Kordower JH et al. Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 2000; 290: 767-773.
11. Palfi S et al. Lentivirally delivered glial cell line-derived neurotrophic factor increases the number of striatal dopaminergic neurons in primate models of nigrostriatal degeneration. J Neurosci 2002; 22: 4942-4954.
12. Kirik D, Rosenblad C, Bjorklund A, Mandel RJ. Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigrostriatal system. J Neurosci 2000; 20: 4686-4700.
13. Wang L et al. Delayed delivery of AAV-GDNF prevents nigral neurodegeneration and promotes functional recovery in a rat model of Parkinson's disease. Gene Therapy 2002; 9: 381-389.
14. McGrath J et al. Adeno-associated viral delivery of GDNF promotes recovery of dopaminergic phenotype following a unilateral 6-hydroxydopamine lesion. Cell Transplant 2002; 11: 215-227.
15. Natsume A et al. Bcl-2 and GDNF delivered by HSV-mediated gene transfer act additively to protect dopaminergic neurons from 6-OHDA-induced degeneration. Exp Neurol 2001; 169: 231-238.
16. Gill SS et al. Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease. Nat Med 2003; 9: 589-595.
17. Georgievska B, Kirik D, Bjorklund A. Aberrant sprouting and downregulation of tyrosine hydroxylase in lesioned nigrostriatal dopamine neurons induced by long-lasting overexpression of glial cell line derived neurotrophic factor in the striatum by lentiviral gene transfer. Exp Neurol 2002; 177: 461-474.
18. Eberhardt O et al. Protection by synergistic effects of adenovirus-mediated X-chromosome-linked inhibitor of apoptosis and glial cell line-derived neurotrophic factor gene transfer in the 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine model of Parkinson's disease. J Neurosci 2000; 20: 9126-9134.
19. Luo J et al. Subthalamic GAD gene therapy in a Parkinson's disease rat model. Science 2002; 298: 425-429.
20. Azzouz M 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 2002; 22: 10302-10312.
21. Kirik D et al. Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer. Proc Natl Acad Sci USA 2002; 99: 4708-4713.
22. Muramatsu S et al. Behavioral recovery in a primate model of Parkinson's disease by triple transduction of striatal cells with adeno-associated viral vectors expressing dopamine-synthesizing enzymes. Hum Gene Ther 2002; 13: 345-354.
23. Shen Y et al. Triple transduction with adeno-associated virus vectors expressing tyrosine hydroxylase, aromatiC-L-amino-acid decarboxylase, and GTP cyclohydrolase I for gene therapy of Parkinson's disease. Hum Gene Ther 2000; 11: 1509-1519.
24. Sun M et al. Correction of a rat model of Parkinson's disease by coexpression of tyrosine hydroxylase and aromatic amino acid decarboxylase from a helper virus-free herpes simplex virus type 1 vector. Hum Gene Ther 2003; 14: 415-424.
25. Lee SH et al. Efficient generation of midbrain and hindbrain neurons from mouse embryonic stem cells. Nat Biotechnol 2000; 18: 675-679.
26. Kim JH et al. Dopamine neurons derived from embryonic stem cells function in an animal model of Parkinson's disease. Nature 2002; 418: 50-56.
27. Bjorklund LM et al. Embryonic stem cells develop into functional dopaminergic neurons after transplantation in a Parkinson rat model. Proc Natl Acad Sci USA 2002; 99: 2344-2349.
28. Shimura H et al. Ubiquitination of a new form of alpha-synuclein by parkin from human brain: implications for Parkinson's disease. Science 2001; 293: 263-269.
29. Bonifati V et al. Mutations in the DJ-1 gene associated with autosomal recessive early-onset parkinsonism. Science 2003; 299: 256-259.
30. Le WD et al. Mutations in NR4A2 associated with familial Parkinson disease. Nat Genet 2003; 33: 85-89.
31. Masliah E et al. Dopaminergic loss and inclusion body formation in alpha-synuclein mice: implications for neurodegenerative disorders. Science 2000; 287: 1265-1269.
32. Lee MK et al. Human alpha-synuclein-harboring familial Parkinson's disease-linked Ala-53 - > Thr mutation causes neurodegenerative disease with alpha-synuclein aggregation in transgenic mice. Proc Natl Acad Sci USA 2002; 99: 8968-8973.
33. Giasson BI et al. Neuronal alpha-synucleinopathy with severe movement disorder in mice expressing A53T human alpha-synuclein. Neuron 2002; 34: 521-533.
34. Richfield EK et al. Behavioral and neurochemical effects of wild-type and mutated human alpha-synuclein in transgenic mice. Exp Neurol 2002; 175: 35-48.
35. Lo Bianco C et al. alpha-Synucleinopathy and selective dopaminergic neuron loss in a rat lentiviral-based model of Parkinson's disease. Proc Natl Acad Sci USA 2002; 99: 10813-10818.
36. Kirik D et al. Nigrostriatal alpha-synucleinopathy induced by viral vector-mediated overexpression of human alpha-synuclein: a new primate model of Parkinson's disease. Proc Natl Acad Sci USA 2003; 100: 2884-2889.
37. Feany MB, Bender WW. A Drosophila model of Parkinson's disease. Nature 2000; 404: 394-398.
38. Auluck PK et al. Chaperone suppression of alpha-synuclein toxicity in a Drosophila model for Parkinson's disease. Science 2002; 295: 865-868.
39. Windisch M et al. Development of a new treatment for Alzheimer's disease and Parkinson's disease using anti-aggregatory beta-synuclein-derived peptides. J Mol Neurosci 2002; 19: 63-69.
40. Petrucelli L et al. Parkin protects against the toxicity associated with mutant alpha-synuclein: proteasome dysfunction selectively affects catecholaminergic neurons. Neuron 2002; 36: 1007-1019.
41. During MJ, Kaplitt MG, Stern MB, Eidelberg D. Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation. Hum Gene Ther 2001; 12: 1589-1591.
42. Xia XG et al. Gene transfer of the JNK interacting protein-1 protects dopaminergic neurons in the MPTP model of Parkinson's disease. Proc Natl Acad Sci USA 2001; 98: 10433-10438.
43. Check E. Regulators split on gene therapy as patient shows signs of cancer. Nature 2002; 419: 545-546.
44. Nakai H et al. AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet 2003; 34: 297-302.
45. Nakamura T et al. Blinded positron emission tomography study of dopamine cell implantation for Parkinson's disease. Ann Neurol 2001; 50: 181-187.
46. Freed CR et al. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N Engl J Med 2001; 344: 710-719.