Intraneural convection enhanced delivery of AAVrh20 for targeting primary sensory neurons

Molecular and Cellular Neuroscience

Volumn 60, Issue , 2014, Pages 72-80

  Pleticha, Josef ^a   Jeng Singh, Christian ^a   Rezek, Rahaf ^a   Zaibak, Manal ^a   Beutler, Andreas S ^a  

^a MAYO CLINIC   (United States)

Author keywords

Adeno associated virus rh20; Convection enhanced delivery; Dorsal root ganglion; Intraneural; Intrathecal; Peripheral nervous system

Indexed keywords

GREEN FLUORESCENT PROTEIN;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ARTICLE; CONVECTION ENHANCED DELIVERY; GENE TRANSFER; NONHUMAN; PERIPHERAL NERVE; PRIORITY JOURNAL; RAT; SCIATIC NERVE; SENSORY NERVE CELL; SEROTYPE; SPINAL GANGLION; THERMODYNAMICS; ADMINISTRATION AND DOSAGE; ANIMAL; CYTOLOGY; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; INJECTION; MALE; METABOLISM; NEURALGIA; PROCEDURES; SPRAGUE DAWLEY RAT;

ANIMALS; CONVECTION; DEPENDOVIRUS; GANGLIA, SPINAL; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; INJECTIONS; MALE; NEURALGIA; RATS; RATS, SPRAGUE-DAWLEY; SCIATIC NERVE; SENSORY RECEPTOR CELLS;

EID: 84900036393     PISSN: 10447431     EISSN: 10959327     Source Type: Journal    
DOI: 10.1016/j.mcn.2014.04.004     Document Type: Article

References (52)

1. Antonijevic I., Mousa S.A., Schäfer M., Stein C. Perineurial defect and peripheral opioid analgesia in inflammation. J. Neurosci. 1995, 15:165-172.
2. Ashtari M., Cyckowski L.L., Monroe J.F., et al. The human visual cortex responds to gene therapy-mediated recovery of retinal function. J. Clin. Invest. 2011, 121:2160-2168. 10.1172/JCI57377.
3. Asokan A., Schaffer D.V., Samulski R.J. The AAV vector toolkit: poised at the clinical crossroads. Mol. Ther. 2012, 20:699-708. 10.1038/mt.2011.287.
4. Beutler A.S. AAV provides an alternative for gene therapy of the peripheral sensory nervous system. Mol. Ther. 2010, 18:670-673. 10.1038/mt.2010.41.
5. Bobo R.H., Laske D.W., Akbasak A., et al. Convection-enhanced delivery of macromolecules in the brain. Proc. Natl. Acad. Sci. U. S. A. 1994, 91:2076-2080.
6. Bowles D.E., McPhee S.W.J., Li C., et al. Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol. Ther. 2012, 20:443-455. 10.1038/mt.2011.237.
7. Cearley C.N., Vandenberghe L.H., Parente M.K., et al. Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol. Ther. 2008, 16:1710-1718. 10.1038/mt.2008.166.
8. Chaplan S.R., Bach F.W., Pogrel J.W., et al. Quantitative assessment of tactile allodynia in the rat paw. J. Neurosci. Methods 1994, 53:55-63.
9. Chen X., Astary G.W., Mareci T.H., Sarntinoranont M. In vivo contrast-enhanced MR imaging of direct infusion into rat peripheral nerves. Ann. Biomed. Eng. 2011, 39:2823-2834. 10.1007/s10439-011-0362-x.
10. Chung J.M., Kim H.K., Chung K. Segmental spinal nerve ligation model of neuropathic pain. Methods Mol. Med. 2004, 99:35-45. 10.1385/1-59259-770-X:035.
11. Eberling J.L., Jagust W.J., Christine C.W., et al. Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 2008, 70:1980-1983. 10.1212/01.wnl.0000312381.29287.ff.
12. Federici T., Boulis N. Gene therapy for peripheral nervous system diseases. Curr. Gene Ther. 2007, 7:239-248.
13. Federici T., Taub J.S., Baum G.R., et al. Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther. 2011, 1-8. 10.1038/gt.2011.130.
14. French J.T., Goins B., Saenz M., et al. Interventional therapy of head and neck cancer with lipid nanoparticle-carried rhenium 186 radionuclide. J. Vasc. Interv. Radiol. 2010, 21:1271-1279. 10.1016/j.jvir.2010.02.027.
15. Fu H., Muenzer J., Samulski R.J., et al. Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol. Ther. 2003, 8:911-917.
16. Gadsden J., McCally C., Hadzic A. Monitoring during peripheral nerve blockade. Curr. Opin. Anaesthesiol. 2010, 23:656-661. 10.1097/ACO.0b013e32833d4f99.
17. Gao G., Vandenberghe L.H., Alvira M.R., et al. Clades of adeno-associated viruses are widely disseminated in human tissues. J. Virol. 2004, 78:6381-6388. 10.1128/JVI.78.12.6381-6388.2004.
18. Gao G., Vandenberghe L.H., Wilson J.M. New recombinant serotypes of AAV vectors. Curr. Gene Ther. 2005, 5:285-297.
19. Gray S.J., Nagabhushan Kalburgi S., McCown T.J., Jude Samulski R. Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. Gene Ther. 2013, 1-10. 10.1038/gt.2012.101.
20. Hadaczek P., Mirek H., Bringas J., et al. Basic fibroblast growth factor enhances transduction, distribution, and axonal transport of adeno-associated virus type 2 vector in rat brain. Hum. Gene Ther. 2004, 15:469-479. 10.1089/10430340460745793.
21. Hadaczek P., Kohutnicka M., Krauze M.T., et al. Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum. Gene Ther. 2006, 17:291-302. 10.1089/hum.2006.17.291.
22. Hollis E.R., Kadoya K., Hirsch M., et al. Efficient retrograde neuronal transduction utilizing self-complementary AAV1. Mol. Ther. 2008, 16:296-301. 10.1038/sj.mt.6300367.
23. Homs J., Ariza L., Pagès G., et al. Schwann cell targeting via intrasciatic injection of AAV8 as gene therapy strategy for peripheral nerve regeneration. Gene Ther. 2011, 18:622-630. 10.1038/gt.2011.7.
24. Jacques S.J., Ahmed Z., Forbes A., et al. AAV8(gfp) preferentially targets large diameter dorsal root ganglion neurones after both intra-dorsal root ganglion and intrathecal injection. Mol. Cell. Neurosci. 2012, 49:464-474. 10.1016/j.mcn.2012.03.002.
25. Kaspar B.K., Lladó J., Sherkat N., et al. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 2003, 301:839-842. 10.1126/science.1086137.
26. Krauze M.T., Saito R., Noble C., et al. Reflux-free cannula for convection-enhanced high-speed delivery of therapeutic agents. J. Neurosurg. 2005, 103:923-929. 10.3171/jns.2005.103.5.0923.
27. Kreimer T., Kiser R., Reader A., et al. Anesthetic efficacy of combinations of 0.5mol/L mannitol and lidocaine with epinephrine for inferior alveolar nerve blocks in patients with symptomatic irreversible pulpitis. J. Endod. 2012, 38:598-603. 10.1016/j.joen.2012.02.016.
28. Lawlor P.A., Bland R.J., Mouravlev A., et al. Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates. Mol. Ther. 2009, 17:1692-1702. 10.1038/mt.2009.170.
29. Leone P., Shera D., McPhee S.W.J., et al. Long-term follow-up after gene therapy for canavan disease. Sci. Transl. Med. 2012, 4:165ra163. 10.1126/scitranslmed.3003454.
30. Lock M., Alvira M., Vandenberghe L.H., et al. Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum. Gene Ther. 2010, 21:1259-1271. 10.1089/hum.2010.055.
31. Lonser R.R., Weil R.J., Morrison P.F., et al. Direct convective delivery of macromolecules to peripheral nerves. J. Neurosurg. 1998, 89:610-615. 10.3171/jns.1998.89.4.0610.
32. Mason M.R.J., Tannemaat M.R., Malessy M.J.A., Verhaagen J. Gene therapy for the peripheral nervous system: a strategy to repair the injured nerve?. Curr. Gene Ther. 2011, 11:75-89.
33. Mastakov M.Y., Baer K., Symes C.W., et al. Immunological aspects of recombinant adeno-associated virus delivery to the mammalian brain. J. Virol. 2002, 76:8446-8454.
34. Mittermeyer G., Christine C.W., Rosenbluth K.H., et al. Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease. Hum. Gene Ther. 2012, 23:377-381. 10.1089/hum.2011.220.
35. Nathwani A.C., Tuddenham E.G.D., Rangarajan S., et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 2011, 365:2357-2365. 10.1056/NEJMoa1108046.
36. Ratliff J.K., Oldfield E.H. Convection-enhanced delivery in intact and lesioned peripheral nerve. J. Neurosurg. 2001, 95:1001-1011. 10.3171/jns.2001.95.6.1001.
37. Rosenbluth K.H., Luz M., Mohr E., et al. Design of an in-dwelling cannula for convection-enhanced delivery. J. Neurosci. Methods 2011, 196:118-123. 10.1016/j.jneumeth.2010.12.022.
38. Samaranch L., Salegio E.A., San Sebastian W., et al. Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum. Gene Ther. 2012, 389:382-389. 10.1089/hum.2011.200.
39. Samaranch L., Salegio E.A., San Sebastian W., et al. Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the CSF of non-human primates. Hum. Gene Ther. 2013, 1-21. 10.1089/hum.2013.005.
40. Snyder B.R., Gray S.J., Quach E.T., et al. Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery. Hum. Gene Ther. 2011, 22:1129-1135. 10.1089/hum.2011.008.
41. Sondhi D., Johnson L., Purpura K., et al. Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis. Hum. Gene Ther. Methods 2012, 23:324-335. 10.1089/hgtb.2012.120.
42. Storek B., Harder N.M., Banck M.S., et al. Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats. Mol. Pain 2006, 2:4. 10.1186/1744-8069-2-4.
43. Storek B., Reinhardt M., Wang C., et al. Sensory neuron targeting by self-complementary AAV8 via lumbar puncture for chronic pain. Proc. Natl. Acad. Sci. U. S. A. 2008, 105:1055-1060. 10.1073/pnas.0708003105.
44. Towne C., Pertin M., Beggah A.T., et al. Recombinant adeno-associated virus serotype 6 (rAAV2/6)-mediated gene transfer to nociceptive neurons through different routes of delivery. Mol. Pain 2009, 5:52. 10.1186/1744-8069-5-52.
45. Tsui B., Suresh S. Ultrasound imaging for regional anesthesia in infants, children, and adolescents: a review of current literature and its application in the practice of extremity and trunk blocks. Anesthesiology 2010, 112:473-492. 10.1097/ALN.0b013e3181c5dfd7.
46. Vulchanova L., Schuster D.J., Belur L.R., et al. Differential adeno-associated virus mediated gene transfer to sensory neurons following intrathecal delivery by direct lumbar puncture. Mol. Pain 2010, 6:31. 10.1186/1744-8069-6-31.
47. White E., Bienemann A., Malone J., et al. An evaluation of the relationships between catheter design and tissue mechanics in achieving high-flow convection-enhanced delivery. J. Neurosci. Methods 2011, 199:87-97. 10.1016/j.jneumeth.2011.04.027.
48. Wolf R., Reader A., Drum M., et al. Anesthetic efficacy of combinations of 0.5m mannitol and lidocaine with epinephrine in inferior alveolar nerve blocks: a prospective randomized, single-blind study. Anesth. Prog. 2011, 58:157-165. 10.2344/11-30.1.
49. Wolfe D., Mata M., Fink D.J. Targeted drug delivery to the peripheral nervous system using gene therapy. Neurosci. Lett. 2012, 527:85-89. 10.1016/j.neulet.2012.04.047.
50. Xu Y., Gu Y., Wu P., et al. Efficiencies of transgene expression in nociceptive neurons through different routes of delivery of adeno-associated viral vectors. Hum. Gene Ther. 2003, 14:897-906. (doi: p:).
51. Yaksh T.L., Rudy T.A. Chronic catheterization of the spinal subarachnoid space. Physiol. Behav. 1976, 17:1031-1036. (doi: 0031-9384(76)90029-9 [pii]).
52. Yin D., Forsayeth J., Bankiewicz K.S. Optimized cannula design and placement for convection-enhanced delivery in rat striatum. J. Neurosci. Methods 2010, 187:46-51. 10.1016/j.jneumeth.2009.12.008.