Gene delivery to the nervous system

Molecular Therapy

Volumn 16, Issue 4, 2008, Pages 640-646

  Schubert, Manfred ^a   Breakefield, Xandra ^b   Federoff, Howard ^c   Frederickson, Robert M ^d   Lowenstein, Pedro R ^e  

^a NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

^c GEORGETOWN UNIVERSITY MEDICAL CENTER   (United States)

^d Molecular Therapy   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA SYNUCLEIN; BETA GLUCURONIDASE; CYCLOPHOSPHAMIDE; FLT3 LIGAND; GANCICLOVIR; GLUTAMATE DECARBOXYLASE; HEAT SHOCK PROTEIN 104; INSULIN RECEPTOR; INTERLEUKIN 10; INTERLEUKIN 4; LENTIVIRUS VECTOR; LEVODOPA; LIPOSOME; MACROGOL; MONOCLONAL ANTIBODY; NERVE GROWTH FACTOR; NEURITE PROMOTING FACTOR; PARKIN; PARVOVIRUS VECTOR; PROENKEPHALIN; SMALL INTERFERING RNA; SOMATOMEDIN C; SUPEROXIDE DISMUTASE; THYMIDINE KINASE; TRANSFERRIN RECEPTOR; TUMOR NECROSIS FACTOR ALPHA;

ABSENCE OF SIDE EFFECTS; ALZHEIMER DISEASE; AMYOTROPHIC LATERAL SCLEROSIS; BLOOD BRAIN BARRIER; BRAIN TUMOR; CELL BASED GENE THERAPY; CLINICAL TRIAL; CONFERENCE PAPER; DEGENERATIVE DISEASE; DRUG EFFICACY; DRUG SAFETY; EXPRESSION VECTOR; FOOD AND DRUG ADMINISTRATION; GENE DELIVERY SYSTEM; GENE TARGETING; GLIOBLASTOMA; GOOD MANUFACTURING PRACTICE; HUMAN; IMMUNE RESPONSE; IN VIVO GENE TRANSFER; LIPOSOMAL GENE DELIVERY SYSTEM; MEDICAL RESEARCH; MENINGOENCEPHALITIS; METACHROMATIC LEUKODYSTROPHY; MOLECULAR CLONING; NEUROLOGIC DISEASE; NEUROMUSCULAR SYNAPSE; NEURONAL CEROID LIPOFUSCINOSIS; NEUROPATHIC PAIN; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PARKINSON DISEASE; PRION DISEASE; RNA INTERFERENCE; STANDARDIZATION; TH2 CELL; VACCINATION; VIRAL GENE DELIVERY SYSTEM;

EID: 41149114159     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2008.42     Document Type: Conference Paper

References (28)

1. Finkelstein, R, Baughman, RW and Steele, FR (2001). Harvesting the neural gene therapy frult. Mol Ther 3: 3-7.
2. Lowenstein, PR Kroeger, KM, Castro, MG and Friedman, TF et al. (2007). Gene therapy for central nervous system disorders. In: Rosenburg, RN, DiMauro, S, Paulson, HL, Ptácek, L, and Nestler, EJ (eds). The Molecular and Genetic Basis of Neurologic and Psychiatric Disease, 4th edn. Lippincott Williams & Wilkins: Philadelphia. pp 87-103.
3. Pulkkanen, KJ and Yla-Herttuala, S (2005). Gene therapy for malignant glioma: Current clinical status. Mol Ther 12: 585-598.
4. Alexander, BL, Ali, RR, Alton, EW, Bainbridge, JW, Braun, S, Cheng, SH et al. (2007). Progress and prospects. 1. Gene therapy clinical trials. Gene Ther 14: 1439-1447.
5. Thomas, CE, Schiedner, G, Kochanek, S, Castro, MG and Lowenstein, PR (2000). Peripheral infection with adenovirus causes unexpected long-term brain inflammation in animals injected intracranially with first-generation, but not with high-capacity, adenovirus vectors: Toward realistic long-term neurological gene therapy for chronic diseases. Proc Natl Acad Sci USA 97: 7482-7487.
6. Lowenstein, PR, Mandel, RJ, Xiong, WD, Kroeger, K and Castro, MG (2007). Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: The role of immunological synapses in understanding the cell biology of neuroimmune interactions. Curr Gene Ther 7: 347-360.
7. Fiandaca, MS, Forsayeth, JR, Dickinson, PJ and Bankiewicz, KS (2008). Image-guided convection-enhanced delivery platform in the treatment of neurological diseases. Neurotherapeutics 5: 123-127.
8. Cearley, CN and Wolfe, JH (2007). A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease. J Neurosci 27: 9928-9940.
9. Barcia, C, Thomas, CE, Curtin, JF, King, GD, Wawrowsky, K, Candolfi, M et al. (2006). In vivo mature immunological synapses forming SMACs mediate clearance of virally infected astrocytes from the brain. J Exp Med 203: 2095-2107.
10. Jakobsson, J and Lundberg, C (2006). Lentiviral vectors for use in the central nervous system. Mol Ther 13: 484-493.
11. Wong, LF, Goodhead, L, Prat, C, Mitrophanous, KA, Kingsman, SM and Mazarakis, ND (2006). Lentivirus-mediated gene transfer to the central nervous system: Therapeutic and research applications. Hum Gene Ther 17:1-9.
12. Mazarakis, ND, Azzouz, M, Rohll, JB, Ellard, FM, Wilkes, FJ, Olsen AL et al. (2001). 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.
13. Mentis, GZ, Gravell, M, Hamilton, R, Shneider, NA, O'Donovan, MJ and Schubert M (2006). Transduction of motor neurons and muscle fibers by intramuscular injection of HIV-1-based vectors pseudotyped with select rabies virus glycoproteins. J Neurosci Methods 157: 208-217.
14. Kaspar, BK Lladó, J, Sherkat N, Rothstein, JD and Gage, FH (2003). Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301: 839-842.
15. Herweijer, H and Wolff, JA (2007). Gene therapy progress and prospects: hydrodynamic gene delivery. Gene Ther 14: 99-107.
16. Ali, S, King, GD, Curtin, JR, Candolfi, M, Xiong, W, Liu, C et al. (2005). Combined immunostimulation and conditional cytotoxic gene therapy provide long-term survival in a large glioma model. Cancer Res 65: 7194-7204.
17. Jiang, H, Gomez-Manzano, C, Aoki, H, Alonso, MM, Kondo, S, McCormick F et al. (2007). Examination of the therapeutic potential of Delta-24-RGD in brain tumor stem cells: Role of autophagic cell death. J Natl Cancer Inst 99:1410-1414.
18. Fulci, G, Breymann, L, Gianni, D, Kurozomi, K, Rhee, SS, Yu, J et al. (2006). Cyclophosphamide enhances glioma virotherapy by inhibiting innate immune responses. Proc Natl Acad Sci USA 103: 12873-12878.
19. Hackett, NR, Redmond, DE, Sondhi, D, Giannaris, EL, Vassallo, E, Stratton, J et al. (2005). Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates. Hum Gene Ther 16: 1484-1503.
20. Glorioso, JC and Fink, DJ (2004). Herpes vector-mediated gene transfer in treatment of diseases of the nervous system. Annu Rev Microbiol 58: 253-271.
21. Tuszynski, MH (2007). Nerve growth factor gene therapy in Alzheimer disease. Alzheimer Dis Assoc Disord 21: 179-189.
22. Federoff, HJ and Bowers, WJ (2005). Immune shaping and the development of Alzheimer's disease vaccines. Sci Aging Knowledge Environ 46 pe35.
23. Bankiewicz, KS, Forsayeth, J, Eberling, JL, Sanchez-Pernaute, R, Pivirotto, P, Bringas, J et al. (2006). Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther 14: 564-570.
24. Kaplitt, MG, Feigin, A, Tang, C, Fitzsimons, HL, Mattis, P, Lawlor, PA et al. (2007). 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.
25. Davidson, BL and Boudreau, RL (2007). RNA interference: A tool for querying nervous system function and an emerging therapy. Neuron 53: 781-788.
26. Levine, BL, Humeau, LM, Boyer, J, MacGregor, RR, Rebello, T, Lu, X et al. (2006). Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA 103: 17372-17377.
27. Biffi, A, Capotondo, A, Fasano, S, del Carro, U, Marchesini, S, Azuma, H et al. (2006). Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest 116: 3070-3082.
28. Paoloni, M and Khanna, C (2008). Translation of new cancer treatments from pet dogs to humans. Nat Rev Cancer 8: 147-156.