Prospects for gene therapy in hearing loss

Journal of Basic and Clinical Physiology and Pharmacology

Volumn 14, Issue 2, 2003, Pages 77-84

  Avraham, Karen B ^a   Raphael, Yehoash ^b  

^a TEL AVIV UNIVERSITY   (Israel)

^b UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

adenoviruses; cochlea; hair cells; inner ear; Math l; retroviruses

Indexed keywords

ACETYLCYSTEINE; ADENOVIRUS VECTOR; AMINOGLYCOSIDE; ANTIAPOPTOTIC AGENT; ANTIOXIDANT; BRAIN DERIVED NEUROTROPHIC FACTOR; CISPLATIN; GENE PRODUCT; GENTAMICIN; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; HELIX LOOP HELIX PROTEIN; NEUROPROTECTIVE AGENT; RECOMBINANT PROTEIN; RETROVIRUS VECTOR; SALICYLIC ACID; SUPEROXIDE DISMUTASE; TRANSCRIPTION FACTOR; TRANSFORMING GROWTH FACTOR BETA; UNCLASSIFIED DRUG;

CELL DEATH; CELL PROTECTION; COCHLEA; GENE; GENE INSERTION; GENE THERAPY; HAIR CELL; HATH1 GENE; HEARING LOSS; HUMAN; INNER EAR; MATH1 GENE; NOISE INJURY; NONHUMAN; OTOTOXICITY; REVIEW; VIRAL GENE DELIVERY SYSTEM;

CELL DEATH; GENE THERAPY; HAIR CELLS; HEARING LOSS; HUMANS;

EID: 0141963359     PISSN: 07926855     EISSN: 21910286     Source Type: Journal    
DOI: 10.1515/JBCPP.2003.14.2.77     Document Type: Article

References (24)

1. Nadol, JB. Hearing Loss. N Engl J Med 1993; 329: 1092–1102.
2. Holley, MC. Application of new biological approaches to stimulate sensory repair and protection. Br Med Bull 2002; 63: 157–169.
3. Raphael, Y. Cochlear pathology, sensory cell death and regeneration. Br Med Bull 2002; 63: 25–38.
4. Lalwani, AK, Jero, J, Mhatre, AN. Developments in cochlear gene therapy. Adv Otorhinolaryngol 2002; 61: 28–33.
5. rd, Hochedlinger, K, Kyba, M, Daley, GQ, Jaenisch, R. Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy. Cell 2002; 109: 17–27.
6. Hacein-Bey-Abina, S, Le Deist, F, Carlier, F, Bouneaud, C, Hue, C, De Villartay, JP, Thrasher, AJ, Wulffraat, N, Sorensen, R, Dupuis-Girod, S, Fischer, A, Davies, EG, Kuis, W, Leiva, L, Cavazzana-Calvo, M. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002; 346: 1185–1193.
7. Weissman, IL. Translating stem and progenitor cell biology to the clinic: barriers and opportunities. Science 2000; 287: 1442–1446.
8. Benvenisty, N. Europe and the stem cell debate. Trends Biotechnol 2002; 20: 183.
9. Gropp, M, Itsykson, P, Singer, O, Ben-Hur, T, Reinhartz, E, Galun, E, Reubinoff, BE,. Stable genetic modification of human embryonic stem cells by lentiviral vectors. Mol Ther 2003; 7: 281–287.
10. Staecker, H, Gabaizadeh, R, Federoff, H, Van De Water, TR. Brain-derived neurotrophic factor gene therapy prevents spiral ganglion degeneration after hair cell loss. Otolaryngol Head Neck Surg 1998; 119:7–13.
11. Suzuki, M, Yagi, M, Brown, JN, Miller, AL, Miller, JM, Raphael, Y. Effect of transgenic GDNF expression on gentamicin-induced cochlear and vestibular toxicity. Gene Ther 2000; 7: 1046–1054.
12. Kawamoto, K, Yagi, M, Stover, T, Kanzaki, S, Raphael, Y. Hearing and hair cells are protected by adenoviral gene therapy with TGF-betal and GDNF. Mol Ther 2003; 7: 484–492.
13. Scarpidis, U, Madnani, D, Shoemaker, C, Fletcher, CH, Kojima, K, Eshraghi, AA, Staecker, H, Lefebvre, P, Malgrange, B, Balkany, TJ, Van De Water, TR. Arrest of apoptosis in auditory neurons: implications for sensorineural preservation in cochlear implantation. Otol Neurotol 2003; 24: 409–417.
14. Ylikoski, J, Xing-Qun, L, Virkkala, J, Pirvola, U. Blockade of c-Jun N-terminal kinase pathway attenuates gentamicin-induced cochlear and vestibular hair cell death. Hear Res 2002; 166: 33–43.
15. Liu, W, Staecker, H, Stupak, H, Malgrange, B, Lefebvre, P, Van De Water, TR. Caspase inhibitors prevent cisplatin-induced apoptosis of auditory sensory cells. NeuroReport 1998; 9: 2609–2614.
16. McFadden, SL, Ding, D, Salvemini, D, Salvi, RJ. M40403, a superoxide dismutase mimetic, protects cochlear hair cells from gentamicin, but not cisplatin toxicity. Toxicol Appl Pharmacol 2003; 186: 46–54.
17. Kopke, RD, Weisskopf, PA, Boone, JL, Jackson, RL, Wester, DC, Hoffer, ME, Lambert, DC, Charon, CC, Ding, DL, McBride, D. Reduction of noise-induced hearing loss using L-NAC and salicylate in the chinchilla. Hear Res 2000; 149: 138–146.
18. Shou, J, Zheng, JL, Gao, WQ. Robust generation of new hair cells in the mature mammalian inner ear by adenoviral expression of Hath I. Mol Cell Neurosci 2003; 23: 169–179.
19. Kawamoto, K, Ishimoto, S, Minoda, R, Brough, DE, Raphael, Y. Mathl gene transfer generates new cochlear hair cells in mature guinea pigs in vivo. J Neurosci 2003; 23: 4395–4400.
20. Forge, A, Li, L, Corwin, JT, Nevill, G. Ultrastructural evidence for hair cell regeneration in the mammalian inner ear. Science 1993; 259: 1616–1619.
21. Warchol, ME, Lambert, PR, Goldstein, BJ, Forge, A, Corwin, JT. Regenerative proliferation in inner ear sensory epithelia from adult guinea pigs and humans. Science 1993; 259: 1619–1622.
22. Wareing, M, Mhatre, AN, Pettis, R, Han, JJ, Haut, T, Pfister, MH, Hong, K, Zheng, WW, Lalwani, AK,. Cationic liposome mediated transgene expression in the guinea pig cochlea. Hear Res 1999; 128: 61–69.
23. Probst, FJ, Fridell, RA, Raphael, Y, Saunders, TL, Wang, A, Liang, Y, Morell, RJ, Touchman, JW, Lyons, RH, Noben-Trauth, K, Friedman, TB, Camper, SA. Correction of deafness in shaker-2 mice by an unconventional myosin in a BAC transgene. Science 1998; 280: 1444–1447.
24. Mburu, P, Mustapha, M, Varela, A, Weil, D, El-Amraoui, A, Holme, RH, Rump, A, Hardisty, RE, Blanchard, S, Coimbra, RS, Perfettini, I, Parkinson, N, Mallon, AM, Glenister, P, Rogers, MJ, Paige, AJ, Moir, L, Clay, J, Rosenthal, A, Liu, XZ, Blanco, G, Steel, KP, Petit, C, Brown, SD,. Defects in whirlin, a PDZ domain molecule involved in stereocilia elongation, cause deafness in the whirler mouse and families with DFNB31. Nat Genet 2003; 34: 421–428.