Current status and prospects of gene therapy for the inner ear

Human Gene Therapy

Volumn 22, Issue 11, 2011, Pages 1311-1322

  Sun, Hong ^a   Huang, Aji ^b   Cao, Shousong ^c,d  

^a CENTRAL SOUTH UNIVERSITY   (China)

^b FUDAN UNIVERSITY SHANGHAI CANCER CENTER   (China)

^c ROSWELL PARK CANCER INSTITUTE   (United States)

^d CENTRAL SOUTH UNIVERSITY   (China)

Author keywords

[No Author keywords available]

Indexed keywords

BRAIN DERIVED NEUROTROPHIC FACTOR; CILIARY NEUROTROPHIC FACTOR; CONNEXIN 26; CONNEXIN 30; CONNEXIN 31; CONNEXIN 43; EPIDERMAL GROWTH FACTOR; FIBROBLAST GROWTH FACTOR; FIBROBLAST GROWTH FACTOR 1; FIBROBLAST GROWTH FACTOR 2; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; INHIBITOR OF APOPTOSIS PROTEIN; NERVE GROWTH FACTOR; NEUROTROPHIN 3; NEUROTROPHIN 4; NEUROTROPHIN 7; OTOSPIRALIN; PLATELET DERIVED GROWTH FACTOR; PROTEIN BCL 2; SCATTER FACTOR; SOMATOMEDIN; TRANSCRIPTION FACTOR; TRANSCRIPTION FACTOR CATH1; TRANSCRIPTION FACTOR HATH1; TRANSCRIPTION FACTOR MATH1; TRANSCRIPTION FACTOR XATH1; TRANSFORMING GROWTH FACTOR; UNCLASSIFIED DRUG; UNINDEXED DRUG; VIRUS VECTOR; X LINKED INHIBITOR OF APOPTOSIS;

CELL REGENERATION; GENE DELIVERY SYSTEM; GENE THERAPY; GENE VECTOR; HUMAN; INNER EAR; INNER EAR DISEASE; MEDICAL RESEARCH; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; REVIEW; VIRAL GENE DELIVERY SYSTEM;

EAR, INNER; GENE THERAPY; GENETIC VECTORS; HUMANS; LABYRINTH DISEASES;

EID: 80052699149     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2010.246     Document Type: Review

References (81)

1. Akin, D., Sturgis, J., Ragheb, K., et al. (2007). Bacteria-mediated- delivery of nanoparticles and cargo into cells. Nat. Nanotechnol. 2, 441-449.
2. Anniko, M., Hellstrom, S., Schmidt, S.H., and Spandow, O. (1989). Toxic effects on inner ear of noxious agents passing-through the round window membrane. Acta Otolaryngol. Suppl. 457, 49-56. (Pubitemid 19079195)
3. Bangham, A.D., Standish, M.M., and Watkins, J.C. (1965). Diffusion-of univalent ions across the lamellae of swollen phospholipids. J. Mol. Biol. 13, 238-252.
4. Bedrosian, J.C., Gratton, M.A., Brigande, J.V., et al. (2006). In vivo-delivery of recombinant viruses to the fetal murine cochlea:-Transduction characteristics and long-term effects on auditory-function. Mol. Ther. 14, 328-335. (Pubitemid 44184963)
5. Bermingham, N.A., Hassan, B.A., Price, S.D., et al. (1999). Math1:-An essential gene for the generation of inner ear hair cells. Science 284, 1837-1841.
6. Bird, P.A., Begg, E.J., Zhang, M., et al. (2007). Intratympanic-versus intravenous delivery of methylprednisolone to cochlear-perilymph. Otol. Neurotol. 28, 1124-1130. (Pubitemid 350191089)
7. Brough, D.E. (2007). Methods of gene therapy for treating disorders-of the ear by administering a vector encoding an-atonal-associated factor. U.S. Patent 2007141029. Available at-www.investorvillage.com/mbthread.asp? mb1195&tid-2586647&showall1 (accessed March 2011).
8. Chandrasekhar, S.S., Rubinstein, R.Y., Kwartler, J.A., et al. (2000). Dexamethasone pharmacokinetics in the inner ear:-Comparison of route of administration and use of facilitating-agents. Otolaryngol. Head Neck Surg. 122, 521-528. (Pubitemid 30210667)
9. Chattopadhyay, D., Mitra, S., and Maitra, A. (2005) A-method using inorganic nanoparticles as non-viral vectors-for gene therapy. Patent WO/2005/123142. Available at-www.wipo.int/pctdb/en/wo.jsp?WO2005123142 (accessed-March 2011).
10. Cohen-Salmon, M., del Castillo, F.J., and Petit, C. (2005). Connexins-responsible for hereditary deafness-the tale unfolds. In Gap Junctions in Development and Disease. E. Winterhager,-ed. (Springer-Verlag, Berlin) pp. 111-134.
11. Cooper, L.B., Chan, D.K., Roediger, F.C., et al. (2006). AAVmediated-delivery of the caspase inhibitor XIAP protects-against cisplatin ototoxicity. Otol. Neurotol. 27, 484-490.
12. Delprat, B., Boulanger, A., Wang, J., et al. (2002). Downregulation-of otospiralin, a novel inner ear protein, causes hair-cell degeneration and deafness. J. Neurosci. 22, 1718-1725. (Pubitemid 35386290)
13. Delprat, B., Ruel, J., Guitton, M.J., et al. (2005). Deafness and-cochlear fibrocyte alterations in mice deficient for the inner ear-protein otospiralin. Mol. Cell. Biol. 25, 847-853. (Pubitemid 40096603)
14. Deveraux, Q.L., Takahashi, R., Salvesen, G.S., and Reed, J.C. (1997). X-linked IAP is a direct inhibitor of cell-death proteases. Nature 388, 300-304. (Pubitemid 27317977)
15. Fujiyoshi, T., Hood, L., and Yoo, T.J. (1994). Restoration of brain-stem auditory-evoked potentials by gene transfer in shiverer-mice. Ann. Otol. Rhinol. Laryngol. 103, 449-456. (Pubitemid 24192812)
16. Goycoolea, M.V., Paparella, M.M., Goldberg, B., and Carpenter,-A.M. (1980). Permeability of the round window membrane in-otitis media. Arch. Otolaryngol. 106, 430-433. (Pubitemid 10071629)
17. Gubbels, S.P., Woessner, D.W., Mitchell, J.C., et al. (2008). Functional auditory hair cells produced in the mammalian-cochlea by in utero gene transfer. Nature 455, 537-541.
18. Hakuba, N., Watabe, K., Hyodo J., et al. (2003). Adenovirusmediated- overexpression of a gene prevents hearing loss and-progressive inner hair cell loss after transient cochlear ischemia-in gerbils. Gene Ther. 10, 426-433. (Pubitemid 36372450)
19. Hanawa, H. (2006).Vector for gene therapy and method of-quantifying target protein in mammal or cultured cells with-the administration of the vector for gene therapy. U.S. Patent-Application 20060223767.
20. Hoang Dinh, E., Ahmad, S., Chang, Q., et al. (2009). Diverse-deafness mechanisms of connexin mutations revealed by-studies using in vitro approaches and mouse models. Brain-Res. 1277, 52-69.
21. Huang, Y., Chi, F., Han, Z., et al. (2009). New ectopic vestibular-hair cell-like cells induced by Math1 gene transfer in postnatal-rats. Brain Res. 1276, 31-38.
22. Izumikawa, M., Minoda, R., Kawamoto, K., et al. (2005). Auditory-hair cell replacement and hearing improvement by Atoh1-gene therapy in deaf mammals. Nat. Med. 11, 271-276. (Pubitemid 40460554)
23. Jero, J., Mhatre, A.N., Tseng, C.J., et al. (2001). Cochlear gene-delivery through an intact round window membrane in-mouse. Hum. Gene Ther. 12, 539-548. (Pubitemid 32511075)
24. Jiang, M., Zhang, Y.Q., He, G.X., and Sun, H. (2007). [Protective-effect of NT-3 gene mediated by hydroxyapatite nanoparticle-on the cochlea of guinea pigs injured by excitotoxicity] [article-in Chinese]. Zhong Nan Da Xue Xue Bao Yi Xue Ban 32,-563-567.
25. Jones, J.M., Montcouquiol, M., Dabdoub, A., et al. (2006). Inhibitors-of differentiation and DNA binding (Ids) regulate-Math1 and hair cell formation during the development of the-organ of Corti. J. Neurosci. 26, 550-558. (Pubitemid 43237120)
26. Kadan, M., Gaziglia, M., and Trapnell, B. (1996). Improved-adenoviral vectors and produced cells. WO 96/18418, PCT/-US95/15947.
27. Kaneda, Y., Oshima, K., Morishita, R., and Kubo, T. (2006). Drug-for auditory dysfunction. U.S. Patent 7390482. Available at-www.freepatentsonline. com/7390482.html (accessed March-2011).
28. Kanzaki, S., Shiotani, A., Inoue, M., et al. (2007). Sendai virus-vector-mediated transgene expression in the cochlea in vivo. Audiol. Neurootol. 12, 119-126. (Pubitemid 46207858)
29. Kawamoto, K., Ishimoto, S., Minoda, R., et al. (2003a). Math1-gene transfer generates new cochlear hair cells in mature-guinea pigs in vivo. J. Neurosci. 23, 4395-4400. (Pubitemid 36706168)
30. Kawamoto, K., Yagi, M., Stover, T., et al. (2003b). Hearing and-hair cells are protected by adenoviral gene therapy with TGFb1-and GDNF. Mol. Ther. 7, 484-492. (Pubitemid 36553417)
31. Kelsell, D.P., Dunlop, J., Stevens, H.P., et al. (1997). Connexin 26-mutations in hereditary non-syndromic sensorineural deafness. Nature 387, 80-83. (Pubitemid 27202653)
32. Kikuchi, T., Adams, J.C., Miyabe, Y., et al. (2000). Potassium ion-recycling pathway via gap junction systems in the mammalian-cochlea and its interruption in hereditary nonsyndromic-deafness. Med. Electron Microsc. 33, 51-56. (Pubitemid 32164777)
33. Kopke, R.D., Wassel, R.A., Mondalek, F., et al. (2006). Magnetic-nanoparticles: Inner ear targeted molecule delivery and middle-ear implant. Audiol. Neurootol. 11, 123-133. (Pubitemid 43168184)
34. Lalwani, A.K., Walsh, B.J., Reilly, P.G., et al. (1996). Development-of in vivo gene therapy for hearing disorders: Introduction-of adeno-associated virus into the cochlea of the guinea-pig. Gene Ther. 3, 588-592. (Pubitemid 26258273)
35. Li, S., and Huang, L. (2000). Nonviral gene therapy: Promises-and challenges. Gene Ther. 7, 31-34. (Pubitemid 30077146)
36. Li, H., Liu, H., and Heller, S. (2003). Pluripotent stem cells from-the adult mouse inner ear. Nat. Med. 9, 1293-1299. (Pubitemid 37279853)
37. Liu, Y.H., Ke, X.M., Qin, Y., et al. (2007). Adeno-associated virusmediated-Bcl-xL prevents aminoglycoside-induced hearing-loss in mice. Chin. Med. J. (Engl.) 120, 1236-1240. (Pubitemid 47232907)
38. Luebke, A.E., Steiger, J.D., Hodges, B.L., and Amalfitano, A. (2001). A modified adenovirus can transfect cochlear hair cells-in vivo without compromising cochlear function. Gene Ther. 8,-789-794. (Pubitemid 32509793)
39. Maiorana, C.R., and Staecker, H. (2005). Advances in inner ear-gene therapy: Exploring cochlear protection and regeneration. Curr. Opin. Otolaryngol. Head Neck Surg. 13, 308-312. (Pubitemid 41339039)
40. McFadden, S.L., Ding, D., Jiang H., et al. (2002). Chinchilla models-of selective cochlear hair cell loss. Hear. Res. 174, 230-238. (Pubitemid 35305049)
41. Miller, J.M., Le Prell, C.G., Prieskorn, D.M., et al. (2007). Delayed-neurotrophin treatment following deafness rescues spiral-ganglion cells from death and promotes regrowth of auditory-nerve peripheral processes: Effects of brain-derived neurotrophic-factor and fibroblast growth factor. J. Neurosci. Res. 85, 1959-1969. (Pubitemid 47051484)
42. Miriszlai, E., Benedeczky, I., Csapo, S., and Bodanszky, H. (1978). The ultrastructure of the round window membrane of-the cat. ORL J. Otorhinolaryngol. Relat. Spec. 40, 111-119. (Pubitemid 9005148)
43. Mori, H., Tabata, Y., Ando, K., et al. (2006). Nucleic acidcontaining-complex. U.S. Patent 20090203768. Available at-www. freepatentsonline.com/y2009/0203768.html (accessed-March 2011).
44. Mulligan, R.C. (1993). The basic science of gene therapy. Science-260, 926-932.
45. Nagy, I., Bodmer, M., Brors, D., and Bodmer, D. (2004). Early-gene expression in the organ of Corti exposed to gentamicin. Hear. Res. 195, 1-8. (Pubitemid 39179877)
46. Nakaizumi, T., Kawamoto, K., Minoda, R., and Raphael, Y. (2004). Adenovirus-mediated expression of brain-derived-neurotrophic factor protects spiral ganglion neurons from-ototoxic damage. Audiol. Neurootol. 9, 135-143. (Pubitemid 38478863)
47. Noushi, F., Richardson, R.T., Hardman, J., et al. (2005). Delivery-of neurotrophin-3 to the cochlea using alginate beads. Otol. Neurotol. 26, 528-533. (Pubitemid 40663890)
48. Okano, T., Nakagawa, T., Kita, T., et al. (2006). Cell-gene delivery-of brain-derived neurotrophic factor to the mouse inner ear. Mol. Ther. 14, 866-871. (Pubitemid 44743126)
49. Oshima, K., Shimamura, M., Mizuno, S., et al. (2004). Intrathecal-injection of HVJ-E containing HGF gene to cerebrospinal fluid-can prevent and ameliorate hearing impairment in rats. FASEB-J. 18, 212-214.
50. Osterman, J.V., Waddell, A., Aposhian, H.V. (1970). DNA and-gene therapy: Uncoating of polyoma pseudovirus in mouse-embryo cells. Proc. Natl. Acad. Sci. U.S.A. 67, 37-40.
51. Petersen, M.B., and Willems, P.J. (2006). Non-syndromic, autosomal-recessive deafness. Clin. Genet. 69, 371-392.
52. Pickles, J.O., Harter, C., and Rebillard, G. (1998). Fibroblast-growth factor receptor expression in outer hair cells of rat-cochlea. Neuroreport 9, 4093-4095. (Pubitemid 29030694)
53. Plontke, S.K., Mynatt, R., Gill, R.M., et al. (2007). Concentration-gradient along the scala tympani after local application of-gentamicin to the round window membrane. Laryngoscope-117, 1191-1198. (Pubitemid 47015668)
54. Plontke, S.K., Biegner, T., Kammerer, B., et al. (2008). Dexamethasone-concentration gradients along scala tympani after-application to the round window membrane. Otol. Neurotol. 29, 401-406. (Pubitemid 351450804)
55. Qasba, P.K., and Aposhian, H.V. (1971). DNA and gene therapy:-Transfer of mouse DNA to human and mouse embryonic cells-by polyoma pseudovirions. Proc. Natl. Acad. Sci. U.S.A. 68,-2345-2349.
56. Raphael, Y. (2002). Cochlear pathology, sensory cell death and-regeneration. Br. Med. Bull. 63, 25-38. (Pubitemid 35175749)
57. Raphael, Y., Frisancho, J.C., and Roessler, B.J. (1996). Adenoviral-mediated gene transfer into guinea pig cochlear cells-in vivo. Neurosci Lett. 207, 137-141. (Pubitemid 26117600)
58. Rejali, D., Lee, V.A, Abrashin, K.A., et al. (2007). Cochlear implants-and ex vivo BDNF gene therapy protect spiral ganglion-neurons. Hear. Res. 228, 180-187. (Pubitemid 46660412)
59. Rozenberg, Y., Medvedkin, V., and Anderson, W.F. (2001). Targeted artificial gene delivery. WO 01/12235, PCT/US00/-22619.
60. Salem, A.K., Searson, P.C., and Leong, K.W. (2003). Multifunctional- nanorods for gene delivery. Nat. Mater. 2, 668-671.
61. Sen, L., and Gambhir, S.S. (2007) A method for noninvasively-and quantitatively monitoring therapeutic and diagnostic-transgene expression induced by ex vivo and in vivo gene-targeting in organs, tissues and cells. Patent WO/2007/-109335. Available at www.wipo.int/pctdb/en/wo.jsp?WO-2007109335 (accessed March 2011).
62. Son, S.J., Bai, X., and Lee, S.B. (2007). Inorganic hollow nanoparticles-and nanotubes in nanomedicine. 1. Drug/gene delivery-applications. Drug Discov. Today 12, 650-656. (Pubitemid 47238640)
63. Staecker, H., Kopke, R., Malgrange, B., et al. (1996). NT-3 and/or-BDNF therapy prevents loss of auditory neurons following-loss of hair cells. Neuroreport 7, 889-894. (Pubitemid 26167971)
64. Staecker, H., Praetorius, M., Baker, K., and Brough, D.E. (2007). Vestibular hair cell regeneration and restoration of balance function-induced bymath1 gene transfer. Otol. Neurotol. 28, 223-231. (Pubitemid 46175126)
65. Stover, T., Yagi, M., and Raphael, Y. (2000). Transduction of the-contralateral ear after adenovirus-mediated cochlear gene-transfer. Gene Ther. 7, 377-383. (Pubitemid 30156549)
66. Sun, H., Jiang, M., Li, G.Y., and Zhu, S.H. (2006). Neurotrophin-3-gene transfection of cochlear cells with hydroxyapatite nanoparticle-vector. Presented at the 34th midwinter meeting of the-Association for Research in Otolaryngology, Baltimore, MD,-February 19-23, 2006. Abstract 372. Available at www-.aro.org/archives/2006/2006-372.html (accessed March 2011).
67. Sun, H., Jiang, M., and Zhu, S.H. (2008). [In vitro and in vivo-studies on hydroxyapatite nanoparticles as a novel vector for-inner ear gene therapy] [article in Chinese]. Zhonghua Er Bi-Yan Hou Tou Jing Wai Ke Za Zhi 43, 51-57.
68. Sun, Y., Tang, W., Chang, Q., et al. (2009). Connexin30 null and-conditional connexin26 null mice display distinct pattern and-time course of cellular degeneration in the cochlea. J. Comp. Neurol. 516, 569-579.
69. Suzuki, M., Yamasoba, T., Suzukawa, K., and Kaga, K. (2003). Adenoviral vector gene delivery via the round window-membrane in guinea pigs. Neuroreport 14, 1951-1955. (Pubitemid 38339767)
70. Tamura, T., Kita, T., Nakagawa, T., et al. (2005). Drug delivery to-the cochlea using PLGA nanoparticles. Laryngoscope 115,-2000-2005. (Pubitemid 44391664)
71. Vagle, K., Wang, W., Vargeese, C., et al. (2008). Lipid nanoparticle-based compositions and methods for the delivery of-biologically active molecules. U.S. Patent 2008/0020058 A1. Available at http://ip.com/ patapp/US20080020058 (accessed-March 2011).
72. Wadell, G., Mei, Y.F., Segerman, A., et al. (2002). Viral vector for-gene therapy. Patent WO/02053759. Available at www. europatentbox.com/patent/ EP1348030B1/abstract/221597-.html (accessed March 2011).
73. Wang, Y., Yang, C., Xu, S., et al. (2007). [Report on the study of-WHO ear and hearing disorders survey protocol in Guizhou-Province][article in Chinese]. Lin Chung Er Bi Yan Hou Tou-Jing Wai Ke Za Zhi 21, 731-734.
74. Wang, J., Menchenton, T., Yin, S., et al. (2008). Over-expression-of X-linked inhibitor of apoptosis protein slows presbycusis in-C57BL/6J mice. Neurobiol. Aging 37, 399-410.
75. Wenzel, G.I., Xia, A., Funk, E., et al. (2007). Helper-dependent- adenovirus-mediated gene transfer into the adult mouse cochlea. Otol. Neurotol. 28, 1100-1108. (Pubitemid 350191086)
76. Yamasoba, T., YaGi, M., Roessler, B.J., et al. (1999). Inner ear-transgene expression after adenoviral vector inoculation in the-endolymphatic sac. Hum. Gene Ther. 10, 769-774. (Pubitemid 29168161)
77. Yu, L., and Matsumoto, K. (2006). Vector for transfection of-eukaryotic cells. U.S. Patent 2006/0074045 A1. Available-at http://ip.com/patapp/ US20060074045 (accessed March-2011).
78. Zaki, M., Arash, H., and Hamidreza, G. (2007). Recombinant-protein polymer vectors for systemic gene delivery. U.S. Patent-2007/0098702. Available at www.freepatentsonline.com/-y2007/0098702.html (accessed March 2011).
79. Zhuo, X.L., Wang, Y., Zhuo, W.L., et al. (2008). Adenoviralmediated-up- regulation of Otos, a novel specific cochlear gene,-decreases cisplatin-induced apoptosis of cultured spiral ligament-fibrocytes via MAPK/mitochondrial pathway. Toxicology-248, 33-38.
80. Zou, J., Saulnter, P., Perrier, T., et al. (2008). Distribution of lipid-nanocapsules in different cochlear cell populations after round-window membrane permeation. J. Biomed. Mater. Res. B. Appl. Biomater. 87B, 10-18.
81. Zou, J., Sood, R., Ranjan, S., et al. (2010). Manufacturing and-in vivo inner ear visualization of MRI traceable liposome-nanoparticles encapsulating gadolinium. J. Nanobiotechnol. 8, 32.