VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model

Nature

Volumn 429, Issue 6990, 2004, Pages 413-417

  Azzouz, Mimoun ^a   Ralph, G Scott ^a   Storkebaum, Erik ^b   Walmsley, Lucy E ^a,c   Mitrophanous, Kyriacos A ^a   Kingsman, Susan M ^a   Carmellet, Peter ^b   Mazarakis, Nicholas D ^a  

^a OXFORD BIOMEDICA UK LTD   (United Kingdom)

^b DEPARTMENT OF PLANT SYSTEMS BIOLOGY   (Belgium)

^c Oxxon Pharmaccines Ltd   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BONE; BRAIN; MUSCLE; NEUROLOGY; PATIENT TREATMENT; TOXICITY;

LENTIVIRAL VECTOR; SPINAL CORD;

CELLS;

COPPER ZINC SUPEROXIDE DISMUTASE; LENTIVIRUS VECTOR; SUPEROXIDE DISMUTASE; UNCLASSIFIED DRUG; VASCULOTROPIN;

MEDICINE;

AMYOTROPHIC LATERAL SCLEROSIS; ANGIOGENESIS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DEATH; DISEASE PREDISPOSITION; DRUG EFFICACY; GENE MUTATION; GENE OVEREXPRESSION; GENETIC ENGINEERING; LIFE EXPECTANCY; MOTOR NEURON DISEASE; MOUSE; NEUROPROTECTION; NONHUMAN; PARALYSIS; PRIORITY JOURNAL; RETROGRADE NERVE FIBER TRANSPORT; SPINAL CORD; SURVIVAL; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; AXONAL TRANSPORT; BRAIN STEM; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HUMANS; INFECTIOUS ANEMIA VIRUS, EQUINE; INJECTIONS, INTRAMUSCULAR; MICE; MICE, TRANSGENIC; MOTOR NEURONS; POINT MUTATION; SPINAL CORD; SUPEROXIDE DISMUTASE; SURVIVAL RATE; TIME FACTORS; TRANSGENES; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

ANIMALIA; LENTIVIRUS;

EID: 2642526164     PISSN: 00280836     EISSN: None     Source Type: Journal    
DOI: 10.1038/nature02544     Document Type: Article

References (27)

1. Mulder, D. W. Clinical limits of amyotrophic lateral sclerosis. Adv. Neurol. 36, 15-22 (1982).
2. Carmeliet, P. Blood vessels and nerves: common signals, pathways and diseases. Nature Rev. Genet. 4, 710-720 (2003).
3. Schratzberger, P. et al. Favorable effect of VEGF gene transfer on ischemic peripheral neuropathy. Nature Med. 6, 405-413 (2000).
4. Jin, K. L., Mao, X. O. & Greenberg, D. A. Vascular endothelial growth factor: direct neuroprotective effect in in vitro ischemia. Proc. Natl Acad. Sci. USA 97, 10242-10247 (2000).
5. Oosthuyse, B. et al. Deletion of the hypoxia-response element in the vascular endothelial growth factor promoter causes motor neuron degeneration. Nature Genet. 28, 131-138 (2001).
6. Lambrechts, D. et al. VEGF is a modifier of amyotrophic lateral sclerosis in mice and humans and protects motoneurons against ischemic death. Nature Genet. 34, 383-394 (2003).
7. Gurney, M. E. et al. Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation. Science 264, 1772-1775 (1994).
8. Bruijn, L. I. et al. ALS-linked SOD1 mutant G85R mediates damage to astrocytes and promotes rapidly progressive disease with SOD1-containing inclusions. Neuron 18, 327-338 (1997).
9. Azzouz, M. et al. Increased motoneuron survival and improved neuromuscular function in transgenic ALS mice after intraspinal injection of an adeno-associated virus encoding Bcl-2. Hum. Mol. Genet. 9, 803-811 (2000).
10. Cleveland, D. W. & Rothstein, J. D. From Charcot to Lou Gehrig: deciphering selective motor neuron death in ALS. Nature Rev. Neurosci. 2, 806-819 (2001).
11. Mazarakis, N. D. et al. Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery. Hum. Mol. Genet. 10, 2109-2121 (2001).
12. Wong, L. F. et al. Transduction patterns of pseudotyped lentiviral vectors in the nervous system. Mol. Ther. 9, 101-111 (2004).
13. Rosen, D. R. Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis. Nature 364, 362 (1993).
14. Ferreirinha, F. et al. Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport. J. Clin. Invest. 113, 231-242 (2004).
15. Sagot, Y., Rosse, T., Vejsada, R., Perrelet, D. & Kato, A. C. Differential effects of neurotrophic factors on motoneuron retrograde labeling in a murine model of motoneuron disease. J. Neurosci. 18, 1132-1141 (1998).
16. Dal Canto, M. C. & Gurney, M. E. Neuropathological changes in two lines of mice carrying a transgene for mutant human Cu,Zn SOD, and in mice overexpressing wild type human SOD: a model of familial amyotrophic lateral sclerosis (FALS). Brain Res. 676, 25-40 (1995).
17. Wang, L. J. et al. Neuroprotective effects of glial cell line-derived neurotrophic factor mediated by an adeno-associated virus vector in a transgenic animal model of amyotrophic lateral sclerosis. J. Neurosci. 22, 6920-6928 (2002).
18. Kaspar, B., Llado, J., Sherkat, N., Rothstein, J. & Gage, F. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301, 839-842 (2003).
19. Fukuda, R. et al. Insulin-like growth factor 1 induces hypoxia-inducible factor 1-mediated vascular endothelial growth factor expression, which is dependent on MAP kinase and phosphatidylinositol 3-kinase signaling in colon cancer cells. J. Biol. Chem. 277, 38205-38211 (2002).
20. Hellstrom, A. et al. Low IGF-I suppresses VEGF-survival signaling in retinal endothelial cells: direct correlation with clinical retinopathy of prematurity. Proc. Natl Acad. Sci. USA 98, 5804-5808 (2001).
21. Mitchell, J. D., Wokke, J. H. & Borasio, G. D. Recombinant human insulin-like growth factor I (rhIGF-I) for amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst. Rev. 3, CD002064 (2002).
22. 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. 22, 10302-10312 (2002).
23. Bienemann, A. S. et al. Long-term replacement of a mutated nonfunctional CNS gene: reversal of hypothalamic diabetes insipidus using an EIAV-based lentiviral vector expressing arginine vasopressin. Mol. Ther. 7, 588-596 (2003).
24. Mitrophanous, K. et al. Stable gene transfer to the nervous system using a non-primate lentiviral vector. Gene Ther. 6, 1808-1818 (1999).
25. Rohll, J. B. et al. Design, production, safety, evaluation, and clinical applications of nonprimate lentiviral vectors. Methods Enzymol. 346, 466-500 (2002).
26. Martin-Rendon, E., White, L. J., Olsen, A., Mitrophanous, K. A. & Mazarakis, N. D. New methods to titrate EIAV-based lentiviral vectors. Mol. Ther. 5, 566-570 (2002).
27. Mohammed, S. I. et al. Effects of the cyclooxygenase inhibitor, piroxicam, on tumor response, apoptosis, and angiogenesis in a canine model of human invasive urinary bladder cancer. Cancer Res. 62, 356-358 (2002).