Long-term transgene expression in the central nervous system using DNA nanoparticles

Molecular Therapy

Volumn 17, Issue 4, 2009, Pages 641-650

  Yurek, David M ^a   Fletcher, Anita M ^a   Smith, George M ^a   Seroogy, Kim B ^b   Ziady, Assem G ^c,d   Molter, Joseph ^d   Kowalczyk, Tomasz H ^e   Padegimas, Linas ^e   Cooper, Mark J ^e  

^a UNIVERSITY OF KENTUCKY   (United States)

^b UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

^c CASE WESTERN RESERVE UNIVERSITY   (United States)

^d CASE WESTERN RESERVE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e NONE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; MACROGOL; NAKED DNA; NANOPARTICLE; PLASMID DNA;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BIOLUMINESCENCE; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DOSE RESPONSE; ENCEPHALITIS; GENE EXPRESSION; GENE OVEREXPRESSION; GENETIC TRANSFECTION; IMMUNOHISTOCHEMISTRY; MALE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; RAT; REPORTER GENE; TRANSGENICS;

ANIMALS; BASE SEQUENCE; BRAIN; DNA; DNA PRIMERS; IMMUNOHISTOCHEMISTRY; IN SITU HYBRIDIZATION; LUCIFERASES; NANOPARTICLES; PLASMIDS; RATS; TRANSDUCTION, GENETIC; TRANSGENES;

ANIMALIA;

EID: 63949084384     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.2     Document Type: Article

References (37)

1. Kirchweger, G (2002). Nanoparticles - the next big thing? Mol Ther 6: 301-302.
2. Thomas, CE, Ehrhardt, A and Kay, MA (2003). Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 4: 346-358.
3. Konstan, MW, Davis, PB, Wagener, JS, Hilliard, KA, Stern, RC, Milgram, LJ et al. (2004). Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution. Hum Gene Ther 15: 1255-1269.
4. Ziady, AG, Gedeon, CR, Miller, T, Quan, W, Payne, JM, Hyatt, SL et al. (2003). Transfection of airway epithelium by stable PEGylated poly-L-lysine DNA nanoparticles in vivo. Mol Ther 8: 936-947.
5. Ziady, AG, Gedeon, CR, Muhammad, O, Stillwell, V, Oette, SM, Fink, TL et al. (2003). Minimal toxicity of stabilized compacted DNA nanoparticles in the murine lung. Mol Ther 8: 948-956.
6. Farjo, R, Skaggs, J, Quiambao, AB, Cooper, MJ and Naash, MI (2006). Efficient non-viral ocular gene transfer with compacted DNA nanoparticles. PLoS ONE 1: E38.
7. Liu, G, Li, D, Pasumarthy, MK, Kowalczyk, TH, Gedeon, CR, Hyatt, SL et al. (2003). Nanoparticles of compacted DNA transfect postmitotic cells. J Biol Chem 278: 32578-32586.
8. Chen, X, Kube, DM, Cooper, MJ and Davis, PB (2008). Cell surface nucleolin serves as receptor for DNA nanoparticles composed of pegylated polylysine and DNA. Mol Ther 16: 333-342.
9. Fink, TL, Klepcyk, PJ, Oette, SM, Gedeon, CR, Hyatt, SL, Kowalczyk, TH et al. (2006). Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles. Gene Ther 13: 1048-1051.
10. Schwartz, B, Benoist, C, Abdallah, B, Rangara, R, Hassan, A, Scherman, D et al. (1996). Gene transfer by naked DNA into adult mouse brain. Gene Ther 3: 405-411.
11. Leone, P, Janson, CG, Bilaniuk, L, Wang, Z, Sorgi, F, Huang, L et al. (2000). Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease. Ann Neurol 48: 27-38.
12. Nimesh, S, Goyal, A, Pawar, V, Jayaraman, S, Kumar, P, Chandra, R et al. (2006). Polyethylenimine nanoparticles as efficient transfecting agents for mammalian cells. J Control Release 110: 457-468.
13. Oh, S, Pluhar, GE, McNeil, EA, Kroeger, KM, Liu, C, Castro, MG et al. (2007). Efficacy of nonviral gene transfer in the canine brain. J Neurosurg 107: 136-144.
14. Thorne, RG and Nicholson, C (2006). In vivo diffusion analysis with quantum dots and dextrans predicts the width of brain extracellular space. Proc Natl Acad Sci USA 103: 5567-5572.
15. Bharali, DJ, Klejbor, I, Stachowiak, EK, Dutta, P, Roy, I, Kaur, N et al. (2005). Organically modified silica nanoparticles: A nonviral vector for in vivo gene delivery and expression in the brain. Proc Natl Acad Sci USA 102: 11539-11544.
16. Lai, SK, Hida, K, Man, ST, Chen, C, Machamer, C, Schroer, TA et al. (2007). Privileged delivery of polymer nanoparticles to the perinuclear region of live cells via a non-clathrin, non-degradative pathway. Biomaterials 28: 2876-2884.
17. Shi, N and Pardridge, WM (2000). Noninvasive gene targeting to the brain. Proc Natl Acad Sci USA 97: 7567-7572.
18. Deroose, CM, Reumers, V, Gijsbers, R, Bormans, G, Debyser, Z, Mortelmans, L et al. (2006). Noninvasive monitoring of long-term lentiviral vector-mediated gene expression in rodent brain with bioluminescence imaging. Mol Ther 14: 423-431.
19. Kugler, S, Meyn, L, Holzmuller, H, Gerhardt, E, Isenmann, S, Schulz, JB et al. (2001). Neuron-specific expression of therapeutic proteins: evaluation of different cellular promoters in recombinant adenoviral vectors. Mol Cell Neurosci 17: 78-96.
20. 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.
21. Eslamboli, A, Georgievska, B, Ridley, RM, Baker, HF, Muzyczka, N, Burger, C et al. (2005). Continuous low-level glial cell line-derived neurotrophic factor delivery using recombinant adeno-associated viral vectors provides neuroprotection and induces behavioral recovery in a primate model of Parkinson's disease. J Neurosci 25: 769-777.
22. Yurek, DM, Fletcher, AM, Smith, GM, Padegimas, L and Cooper, MJ (2008). Nano particle gene therapy in an animal model of Parkinson's disease: neuroprotection and expression of GDNF in the nigrostriatal pathway. Mol Ther 16 (suppl. 1): S196
23. Gill, SS, Patel, NK, Hotton, GR, O'Sullivan, K, McCarter, R, Bunnage, M et al. (2003). Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease. Nat Med 9: 589-595.
24. Slevin, JT, Gerhardt, GA, Smith, CD, Gash, DM, Kryscio, R and Young, B (2005). Improvement of bilateral motor functions in patients with Parkinson disease through the unilateral intraputaminal infusion of glial cell line-derived neurotrophic factor. J Neurosurg 102 216-222.
25. Slevin, JT, Gash, DM, Smith, CD, Gerhardt, GA, Kryscio, R, Chebrolu, H et al. (2007). Unilateral intraputamenal glial cell line-derived neurotrophic factor in patients with Parkinson disease: Response to 1 year of treatment and 1 year of withdrawal. J Neurosurg 106: 614-620.
26. Love, S, Plaha, P, Patel, NK, Hotton, GR, Brooks, DJ and Gill, SS (2005). Glial cell line-derived neurotrophic factor induces neuronal sprouting in human brain. Nat Med 11: 703-704.
27. Lang, AE, Gill, S, Patel, NK, Lozano, A, Nutt, JG, Penn, R et al. (2006). Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease. Ann Neurol 59: 459-466.
28. Morrison, PF, Lonser, RR and Oldfield, EH (2007). Convective delivery of glial cell line-derived neurotrophic factor in the human putamen. J Neurosurg 107: 74-83.
29. Salvatore, MF, Ai, Y, Fischer, B, Zhang, AM, Grondin, RC, Zhang, Z et al. (2006). Point source concentration of GDNF may explain failure of phase II clinical trial. Exp Neurol 202: 497-505.
30. Choi-Lundberg, DL, Lin, Q, Schallert, T, Crippens, D, Davidson, BL, Chang, YN et al. (1998). Behavioral and cellular protection of rat dopaminergic neurons by an adenoviral vector encoding glial cell line-derived neurotrophic factor. Exp Neurol 154: 261-275.
31. Mandel, RJ, Snyder, RO and Leff, SE (1999). Recombinant adeno-associated viral vector-mediated glial cell line-derived neurotrophic factor gene transfer protects nigral dopamine neurons after onset of progressive degeneration in a rat model of Parkinson's disease. Exp Neurol 160: 205-214.
32. Kordower, JH, Emborg, ME, Bloch, J, Ma, SY, Chu, Y, Leventhal, L et al. (2000). Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 290 767-773.
33. Kirik, D, Rosenblad, C, Bjorklund, A and Mandel, RJ (2000). 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 20: 4686-4700.
34. Sambrook, J, Fritsch, EF and Maniatis, T (1989). Molecular Cloning: A Laboratory Manual. Cold Spring Harbor Laboratory Press: Cold Spring Harbor, NY.
35. Gill, DR, Smyth, SE, Goddard, CA, Pringle, IA, Higgins, CF, Colledge, WH et al. (2001). Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter. Gene Ther 8: 1539-1546.
36. Baumgartner, BJ and Shine, HD (1997). Targeted transduction of CNS neurons with adenoviral vectors carrying neurotrophic factor genes confers neuroprotection that exceeds the transduced population. J Neurosci 17: 6504-6511.
37. Baumgartner, BJ and Shine, HD (1998). Permanent rescue of lesioned neonatal motoneurons and enhanced axonal regeneration by adenovirus-mediated expression of glial cell-line-derived neurotrophic factor. J Neurosci Res 54: 766-777.