Specific retrograde transduction of spinal motor neurons using lentiviral vectors targeted to presynaptic NMJ receptors

Molecular Therapy

Volumn 22, Issue 7, 2014, Pages 1285-1298

  Eleftheriadou, I ^a   Trabalza, A ^a   Ellison, S M ^a   Gharun, K ^a,b   Mazarakis, N D ^a  

^a HAMMERSMITH HOSPITAL   (United Kingdom)

^b UNIVERSITY OF FREIBURG   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CELL RECEPTOR; COXSACKIE VIRUS AND ADENOVIRUS RECEPTOR; LENTIVIRUS VECTOR; PRESYNAPTIC RECEPTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; BINDING AFFINITY; CONFERENCE PAPER; CONTROLLED STUDY; EMBRYO; GENE TRANSFER; IN VITRO STUDY; LEG MUSCLE; LUMBAR SPINE; MICROFLUIDICS; MOTONEURON; MOUSE; NERVE ENDING; NERVE FIBER; NEUROMUSCULAR SYNAPSE; NEUROTROPISM; NONHUMAN; PRESYNAPTIC NERVE; RAT; SPINAL NERVE; VIRAL GENE DELIVERY SYSTEM; ANIMAL; CELL LINE; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HUMAN; LENTIVIRINAE; METABOLISM; PC12 CELL LINE; PROCEDURES; TUMOR CELL LINE;

ADENOVIRIDAE; COXSACKIEVIRUS; MUS;

ANIMALS; CELL LINE; CELL LINE, TUMOR; GENETIC VECTORS; HUMANS; LENTIVIRUS; MICE; MOTOR NEURONS; NEUROMUSCULAR JUNCTION; PC12 CELLS; RATS; RECEPTORS, PRESYNAPTIC; TRANSDUCTION, GENETIC;

EID: 84903776107     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.49     Document Type: Conference Paper

References (40)

1. Davidson, BL and Breakefield, XO (2003). Viral vectors for gene delivery to the nervous system. Nat Rev Neurosci 4: 353-364. (Pubitemid 37280174
2. 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. (Pubitemid 43102718
3. 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. (Pubitemid 32998823
4. 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. (Pubitemid 44339638
5. Azzouz, M, Le, T, Ralph, GS, Walmsley, L, Monani, UR, Lee, DC et al. (2004). Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. J Clin Invest 114: 1726-1731. (Pubitemid 40385528
6. Azzouz, M, Ralph, GS, Storkebaum, E, Walmsley, LE, Mitrophanous, KA, Kingsman, SM et al. (2004). VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature 429: 413-417. (Pubitemid 38715132
7. Ralph, GS, Radcliffe, PA, Day, DM, Carthy, JM, Leroux, MA, Lee, DC et al. (2005). Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med 11: 429-433. (Pubitemid 40562334
8. Verhoeyen, E and Cosset, FL (2004). Surface-engineering of lentiviral vectors. J Gene Med 6 Suppl 1: S83-S94. (Pubitemid 40228019
9. Yang, L, Bailey, L, Baltimore, D and Wang, P (2006). Targeting lentiviral vectors to specific cell types in vivo. Proc Natl Acad Sci USA 103: 11479-11484. (Pubitemid 44182477
10. Joo, KI and Wang, P (2008). Visualization of targeted transduction by engineered lentiviral vectors. Gene Ther 15: 1384-1396
11. Lei, Y, Joo, KI and Wang, P (2009). Engineering fusogenic molecules to achieve targeted transduction of enveloped lentiviral vectors. J Biol Eng 3: 8
12. Lei, Y, Joo, KI, Zarzar, J, Wong, C and Wang, P (2010). Targeting lentiviral vector to specific cell types through surface displayed single chain antibody and fusogenic molecule. Virol J 7: 35
13. Herreros, J, Ng, T and Schiavo, G (2001). Lipid rafts act as specialized domains for tetanus toxin binding and internalization into neurons. Mol Biol Cell 12: 2947-2960. (Pubitemid 33062953
14. Lalli, G and Schiavo, G (2002). Analysis of retrograde transport in motor neurons reveals common endocytic carriers for tetanus toxin and neurotrophin receptor p75NTR. J Cell Biol 156: 233-239. (Pubitemid 34839905
15. Wong, LF, Azzouz, M, Walmsley, LE, Askham, Z, Wilkes, FJ, Mitrophanous, KA et al. (2004). Transduction patterns of pseudotyped lentiviral vectors in the nervous system. Mol Ther 9: 101-111. (Pubitemid 38208333
16. Soudais, C, Laplace-Builhe, C, Kissa, K and Kremer, EJ (2001). Preferential transduction of neurons by canine adenovirus vectors and their efficient retrograde transport in vivo. FASEB J 15: 2283-2285
17. Salinas, S, Bilsland, LG, Henaff, D, Weston, AE, Keriel, A, Schiavo, G et al. (2009). CARassociated vesicular transport of an adenovirus in motor neuron axons. PLoS Pathog 5: E1000442
18. Ch'ng, TH and Enquist, LW (2005). Neuron-To-cell spread of pseudorabies virus in a compartmented neuronal culture system. J Virol 79: 10875-10889. (Pubitemid 41170630
19. Park, JW, Vahidi, B, Taylor, AM, Rhee, SW and Jeon, NL (2006). Microfluidic culture platform for neuroscience research. Nat Protoc 1: 2128-2136. (Pubitemid 46773335
20. Deinhardt, K, Salinas, S, Verastegui, C, Watson, R, Worth, D, Hanrahan, S et al. (2006). Rab5 and Rab7 control endocytic sorting along the axonal retrograde transport pathway. Neuron 52: 293-305. (Pubitemid 44548343
21. Schiavo, G, Matteoli, M and Montecucco, C (2000). Neurotoxins affecting neuroexocytosis. Physiol Rev 80: 717-766. (Pubitemid 30164948
22. Tissenbaum, HA and Parry, DJ (1991). The effect of partial denervation of tibialis anterior (TA) muscle on the number and sizes of motorneurons in TA motornucleus of normal and dystrophic (C57BL dy2j/dy2j) mice. Can J Physiol Pharmacol 69: 1769-1773
23. Staiger, JF and Nurnberger, F (1990). Columnar Arrangement of Lumbar Motorneurons Innervating a Pair of Antagonistically Acting Leg Muscles in the Rat. Zeitschrift fer mikroskopisch-Anatomische Forschung 104: 79-86. (Pubitemid 20160681
24. Carpentier, DC, Vevis, K, Trabalza, A, Georgiadis, C, Ellison, SM, Asfahani, RI et al. (2012). Enhanced pseudotyping efficiency of HIV-1 lentiviral vectors by a rabies/vesicular stomatitis virus chimeric envelope glycoprotein. Gene Ther 19: 761-774
25. Kato, S, Kobayashi, K, Inoue, K, Kuramochi, M, Okada, T, Yaginuma, H et al. (2011). A lentiviral strategy for highly efficient retrograde gene transfer by pseudotyping with fusion envelope glycoprotein. Hum Gene Ther 22: 197-206
26. Lavillette, D, Russell, SJ and Cosset, FL (2001). Retargeting gene delivery using surface-engineered retroviral vector particles. Curr Opin Biotechnol 12: 461-466. (Pubitemid 32972170
27. Waehler, R, Russell, SJ and Curiel, DT (2007). Engineering targeted viral vectors for gene therapy. Nat Rev Genet 8: 573-587. (Pubitemid 47077278
28. Morizono, K, Xie, Y, Ringpis, GE, Johnson, M, Nassanian, H, Lee, B et al. (2005). Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection. Nat Med 11: 346-352. (Pubitemid 40460565
29. Anliker, B, Abel, T, Kneissl, S, Hlavaty, J, Caputi, A, Brynza, J et al. (2010). Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors. Nat Methods 7: 929-935
30. Funke, S, Maisner, A, Muhlebach, MD, Koehl, U, Grez, M, Cattaneo, R et al. (2008). Targeted cell entry of lentiviral vectors. Mol Ther 16: 1427-1436
31. Funke, S, Schneider, IC, Glaser, S, Muhlebach, MD, Moritz, T, Cattaneo, R et al. (2009). Pseudotyping lentiviral vectors with the wild-Type measles virus glycoproteins improves titer and selectivity. Gene Ther 16: 700-705
32. Enkirch, T, Kneissl, S, Hoyler, B, Ungerechts, G, Stremmel, W, Buchholz, CJ et al. (2013). Targeted lentiviral vectors pseudotyped with the Tupaia paramyxovirus glycoproteins. Gene Ther 20: 16-23
33. Ginty, DD and Segal, RA (2002). Retrograde neurotrophin signaling: Trk-ing along the axon. Curr Opin Neurobiol 12: 268-274. (Pubitemid 34615751
34. Lalli, G, Gschmeissner, S and Schiavo, G (2003). Myosin Va and microtubule-based motors are required for fast axonal retrograde transport of tetanus toxin in motor neurons. J Cell Sci 116(Pt 22): 4639-4650. (Pubitemid 37461618
35. Restani, L, Novelli, E, Bottari, D, Leone, P, Barone, I, Galli-Resta, L et al. (2012). Botulinum neurotoxin A impairs neurotransmission following retrograde transynaptic transport. Traffic 13: 1083-1089
36. Bohnert, S and Schiavo, G (2005). Tetanus toxin is transported in a novel neuronal compartment characterized by a specialized pH regulation. J Biol Chem 280: 42336-42344. (Pubitemid 43023205
37. Deinhardt, K, Berninghausen, O, Willison, HJ, Hopkins, CR and Schiavo, G (2006). Tetanus toxin is internalized by a sequential clathrin-dependent mechanism initiated within lipid microdomains and independent of epsin1. J Cell Biol 174: 459-471. (Pubitemid 44156775
38. Wade, A, Thomas, C, Kalmar, B, Terenzio, M, Garin, J, Greensmith, L et al. (2012). Activated leukocyte cell adhesion molecule modulates neurotrophin signaling. J Neurochem 121: 575-586
39. Trabalza, A, Georgiadis, C, Eleftheriadou, I, Hislop, JN, Ellison, SM, Karavassilis, ME et al. (2013). Venezuelan equine encephalitis virus glycoprotein pseudotyping confers neurotropism to lentiviral vectors. Gene Ther 20: 723-732
40. Benkhelifa-Ziyyat, S, Besse, A, Roda, M, Duque, S, Astord, S, Carcenac, R et al. (2013). Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. Mol Ther 21: 282-290.