Increased encapsulated cell biodelivery of nerve growth factor in the brain by transposon-mediated gene transfer

Gene Therapy

Volumn 19, Issue 10, 2012, Pages 1010-1017

  Fjord Larsen, L ^a   Kusk, P ^a   Emerich, D F ^b   Thanos, C ^c   Torp, M ^a   Bintz, B ^c   Tornoe, J ^a   Johnsen, A H ^d   Wahlberg, L U ^b  

^a NsGene AS   (Denmark)

^b NsGene Inc   (United States)

^c CytoSolv Inc   (United States)

^d Department of Clinical Biochemistry ^*  (Denmark)

Author keywords

Cholinergic neurons; Encapsulated cell biodelivery; NGF; Preclinical validation; Rat QA model; Sleeping beauty transposon

Indexed keywords

QUINOLINIC ACID; RECOMBINANT NERVE GROWTH FACTOR;

ALZHEIMER DISEASE; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN; CELL ENCAPSULATION; CELL LINE; COGNITIVE DEFECT; CONTROLLED STUDY; CORPUS STRIATUM; DEVICE SAFETY; DNA MODIFICATION; DRUG DELIVERY SYSTEM; DRUG DOSE ESCALATION; DRUG POTENCY; GENE EXPRESSION; HUMAN; HUMAN CELL; IMPLANTATION; MALE; NONHUMAN; PRIORITY JOURNAL; RAT; TRANSPOSON;

ALZHEIMER DISEASE; ANIMALS; BRAIN; CAPSULES; CELL LINE, TRANSFORMED; DNA TRANSPOSABLE ELEMENTS; FEMALE; GENE EXPRESSION; HUMANS; NERVE DEGENERATION; NERVE GROWTH FACTOR; QUINOLINIC ACID; RATS; RATS, SPRAGUE-DAWLEY; TRANSFECTION;

ANIMALIA; RATTUS;

EID: 84867403458     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2011.178     Document Type: Article

References (37)

1. Jonhagen ME. Nerve growth factor treatment in dementia. Alzheimer Dis Assoc Disord 2000; 14 (Suppl 1): S31 -S38. (Pubitemid 30406770)
2. Rosenberg MB, Friedmann T, Robertson RC, Tuszynski M, Wolff JA, Breakefield XO et al. Grafting genetically modified cells to the damaged brain: restorative effects of NGF expression. Science 1988; 242: 1575-1578. (Pubitemid 19023703)
3. Koliatsos VE, Clatterbuck RE, Nauta HJ, Knusel B, Burton LE, Hefti FF et al. Human nerve growth factor prevents degeneration of basal forebrain cholinergic neurons in primates. Ann Neurol 1991; 30: 831 -840.
4. Kordower JH, Winn SR, Liu YT, Mufson EJ, Sladek Jr JR, Hammang JP et al. The aged monkey basal forebrain: rescue and sprouting ofaxotomized basalforebrain neurons after grafts ofencapsulated cells secreting human nerve growth factor. Proc Natl Acad Sci USA 1994; 91: 10898 -10902. (Pubitemid 24349592)
5. Martinez-Serrano A, Fischer W, Soderstrom S, Ebendal T, Bjorklund A. Long-term functional recovery from age-induced spatial memory impairments by nerve growth factor genetransfer to the rat basal forebrain. Proc Natl Acad Sci USA 1996; 93: 6355-6360. (Pubitemid 26230309)
6. Conner JM, Darracq MA, Roberts J, Tuszynski MH. Nontropic actions of neurotrophins: subcortical nerve growth factor gene delivery reverses age-related degeneration of primate cortical cholinergic innervation. Proc Natl Acad Sci USA 2001; 98: 1941 -1946. (Pubitemid 32165602)
7. Tuszynski MH, Grill R, Jones LL, McKay HM, Blesch A. Spontaneous and augmented growth of axons in the primate spinal cord: effects of local injury and nerve growth factor-secreting cell grafts. J Comp Neurol 2002; 449: 88-101. (Pubitemid 34625696)
8. Li LY, Li JT, Wu QY, Li J, Feng ZT, Liu S et al. Transplantation of NGF-gene-modified bone marrow stromal cells into a rat model of Alzheimer' disease. J Mol Neurosci 2008; 34: 157-163.
9. Salehi A, Delcroix JD, Swaab DF. Alzheimer's disease and NGF signaling. J Neural Transm 2004; 111: 323-345. (Pubitemid 38316609)
10. Lindvall O, Wahlberg LU. Encapsulated cell biodelivery of GDNF: a novel clinical strategy for neuroprotection and neuroregeneration in Parkinson's disease? Exp Neurol 2008; 209: 82-88.
11. Hoffman D, Breakefield XO, Short MP, Aebischer P. Transplantation of a polymer-encapsulated cell line genetically engineered to release NGF. Exp Neurol 1993; 122: 100-106. (Pubitemid 23242275)
12. Emerich DF, Winn SR, Harper J, Hammang JP, Baetge EE, Kordower JH. Implants of polymer-encapsulated human NGF-secreting cells in the nonhuman primate: rescue and sprouting of degenerating cholinergic basal forebrain neurons. J Comp Neurol 1994; 349: 148-164. (Pubitemid 24328525)
13. Lindner MD, Kearns CE, Winn SR, Frydel B, Emerich DF. Effects of intraventricular encapsulated hNGF-secreting fibroblasts in aged rats.CellTransplant 1996;5:205-223. (Pubitemid 26096181)
14. Winn SR, Hammang JP, Emerich DF, Lee A, Palmiter RD, Baetge EE. Polymer-encapsulated cells genetically modified to secrete human nerve growth factor promote the survival ofaxotomized septal cholinergic neurons. Proc Natl Acad Sci USA 1994; 91: 2324-2328. (Pubitemid 24092096)
15. Fjord-Larsen L, Kusk P, Tornoe J, Juliusson B, Torp M, Bjarkam CR et al. Long-term delivery of nerve growth factor by encapsulated cell biodelivery in the Gottingen minipig basal forebrain. Mol Ther 2010; 18: 2164-2172.
16. Dunn KC, Aotaki-Keen AE, Putkey FR, Hjelmeland LM. ARPE-19, a human retinal pigment epithelial cell line with differentiated properties. Exp Eye Res 1996; 62: 155-169.
17. Ivics Z, Hackett PB, Plasterk RH, Izsvak Z. Molecular reconstruction of sleeping beauty, a Tc1-like transposon from fish, and its transposition in human cells. Cell 1997; 91: 501 -510. (Pubitemid 27508239)
18. Ivics Z, IzsvakZ. The expanding universe of transposon technologies forgene and cell engineering. Mob DNA 2010; 1: 25.
19. Hui EK, Wang PC, Lo SJ. Strategies for cloning unknown cellular flanking DNA sequences from foreign integrants. Cell Mol Life Sci 1998; 54: 1403-1411. (Pubitemid 29015299)
20. Klein RL, Hirko AC, Meyers CA, Grimes JR, Muzyczka N, Meyer EM. NGF gene transfer to intrinsic basal forebrain neurons increases cholinergic cell size and protects from age-related, spatial memory deficits in middle-aged rats. Brain Res 2000; 875: 144-151.
21. Bishop KM, Hofer EK, Mehta A, Ramirez A, Sun L, Tuszynski M et al. Therapeutic potential of CERE-110 (AAV2-NGF): targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neurons. Exp Neurol 2008; 211: 574-584.
22. Kordower JH, Chen EY, Mufson EJ, Winn SR, Emerich DF. Intrastriatal implants of polymer encapsulated cells genetically modified to secrete human nerve growth factor: trophic effects upon cholinergic and noncholinergic striatal neurons. Neuroscience 1996; 72: 63 - 77. (Pubitemid 26092377)
23. Frim DM, Uhler TA, Short MP, Ezzedine ZD, Klagsbrun M, Breakefield XO et al. Effects of biologically delivered NGF, BDNF and bFGF on striatal excitotoxic lesions. Neuroreport 1993; 4: 367-370. (Pubitemid 23116724)
24. Emerich DF, Hammang JP, Baetge EE, Winn SR. Implantation of polymer-encapsulated human nerve growth factor-secreting fibroblasts attenuates the behavioral and neuropathological consequences ofquinolinic acid injections into rodent striatum. Exp Neurol 1994; 130: 141 -150.
25. Emerich DF, Mooney DJ, Storrie H, Babu RS, Kordower JH. Injectable hydrogels providing sustained delivery ofvascular endothelial growth factor are neuropro-tective in a rat model of Huntington's disease. Neurotox Res 2010; 17: 66-74.
26. Jørgensen JR, Emerich DF, Thanos C, Thompson LH, Torp M, Bintz B et al. Lentiviral delivery of Meteorin protects striatal neurons against excitotoxicity and reverses motor deficits in the quinolinic acid rat model. Neurobiol Dis 2011; 41: 160-168.
27. Mullen RJ, Buck CR, Smith AM. NeuN, a neuronal specific nuclear protein in vertebrates. Development 1992; 116: 201 -211.
28. Grabundzija I, Irgang M, Mates L, Belay E, Matrai J, Gogol-Doring A et al. Comparative analysis oftransposable element vector systems in human cells.Mol Ther2010; 18: 1200-1209.
29. Ting KK, Brew BJ, Guillemin GJ. Effect of quinolinic acid on human astrocytes morphology and functions: implications in Alzheimer's disease. J Neuroinflamma-tion 2009; 6: 36.
30. Rahman A,Ting K,Cullen KM,Braidy N, BrewBJ,Guillemin GJ.Theexcitotoxin quinolinic acid induces tau phosphorylation in human neurons. PLoS One 2009; 4: e6344.
31. Montero CN, Hefti F. Rescue of lesioned septal cholinergic neurons by nerve growth factor: specificity and requirement for chronic treatment. J Neurosci 1988; 8: 2986-2999.
32. Niewiadomska G, Komorowski S, Baksalerska-Pazera M. Amelioration ofcholiner-gic neurons dysfunction in aged rats depends on the continuous supply of NGF. Neurobiol Aging 2002; 23: 601 -613. (Pubitemid 34495465)
33. Good PF, Werner P, Hsu A, Olanow CW, Perl DP. Evidence of neuronal oxidative damage in Alzheimer's disease. Am J Pathol 1996; 149: 21 -28. (Pubitemid 26230974)
34. Butterfield DA, Perluigi M, Sultana R. Oxidative stress in Alzheimer's disease brain: new insights from redox proteomics. Eur J Pharmacol 2006; 545: 39-50. (Pubitemid 44215876)
35. Heneka MT, O'Banion MK, Terwel D, Kummer MP. Neuroinflammatory processes in Alzheimer's disease. J Neural Transm 2010; 117: 919-947.
36. Fjord-Larsen L, Johansen JL, Kusk P, Tornøe J, Gronborg M, Rosenblad C et al. Efficient in vivo protection of nigral dopaminergic neurons by lentiviral gene transfer of a modified Neurturin construct. Exp Neurol 2005; 195: 49-60. (Pubitemid 41116813)
37. Demeure K, Quinton L, Gabelica V, De PE. Rational selection of the optimum MALDI matrix for top-down proteomics by in-source decay. Anal Chem 2007; 79: 8678-8685. (Pubitemid 350171379)