Early postnatal virus inoculation into the scala media achieved extensive expression of exogenous green fluorescent protein in the inner ear and preserved auditory brainstem response thresholds

Journal of Gene Medicine

Volumn 15, Issue 3-4, 2013, Pages 123-133

  Wang, Yunfeng ^a,c   Sun, Yu ^b   Chang, Qing ^c   Ahmad, Shoeb ^c   Zhou, Binfei ^c   Kim, Yeunjung ^c   Li, Huawei ^a   Lin, Xi ^c  

^a FUDAN UNIVERSITY   (China)

^b HUAZHONG UNIVERSITY OF SCIENCE AND TECHNOLOGY   (China)

^c EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Cochlea; Exogenous gene expression; Gene therapy; Hearing sensitivity; Mouse; Scala media; Virus

Indexed keywords

CONNEXIN 26; GREEN FLUORESCENT PROTEIN; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ARTICLE; AUDITORY THRESHOLD; CELL TYPE; COCHLEA; CONTROLLED STUDY; CYTOMEGALOVIRUS; EARLY INTERVENTION; ENDOLYMPH; EVOKED BRAIN STEM AUDITORY RESPONSE; FEMALE; GAP JUNCTION; GENE EXPRESSION; HAIR CELL; HEARING ACUITY; HISTOLOGY; INNER EAR; INOCULATION; LENTIVIRINAE; LOSS OF FUNCTION MUTATION; MALE; MOUSE; NONHUMAN; POSTNATAL DEVELOPMENT; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN EXPRESSION; TRANSGENE;

ANIMALS; AUDITORY THRESHOLD; COCHLEAR DUCT; DEPENDOVIRUS; EVOKED POTENTIALS, AUDITORY, BRAIN STEM; GENE EXPRESSION REGULATION; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEARING LOSS, SENSORINEURAL; HISTOLOGICAL TECHNIQUES; LENTIVIRUS; MICE; TRANSGENES;

ADENO-ASSOCIATED VIRUS; CYTOMEGALOVIRUS; EPITONIUM; LENTIVIRUS; MUS;

EID: 84876698758     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.2701     Document Type: Article

References (40)

1. Sundstrom RA, Van Laer L, Van Camp G, et al. Autosomal recessive nonsyndromic hearing loss. Am J Med Genet 1999; 89: 123-129.
2. Denoyelle F, Marlin S, Weil D, et al. Clinical features of the prevalent form of childhood deafness, DFNB1, due to a connexin-26 gene defect: implications for genetic counselling. Lancet 1999; 353: 1298-1303.
3. Wang Y, Chang Q, Tang W, et al. Targeted connexin26 ablation arrests postnatal development of the organ of Corti. Biochem Biophys Res Commun 2009; 385: 33-37.
4. Wangemann P, Itza EM, Albrecht B, et al. Loss of KCNJ10 protein expression abolishes endocochlear potential and causes deafness in Pendred syndrome mouse model. BMC Med 2004; 2: 30.
5. Sadanaga M, Morimitsu T. Development of endocochlear potential and its negative component in mouse cochlea. Hear Res 1995; 89: 155-161.
6. Wareing M, Mhatre AN, Pettis R, et al. Cationic liposome mediated transgene expression in the guinea pig cochlea. Hear Res 1999; 128: 61-69.
7. Zheng JL, Gao WQ. Overexpression of Math1 induces robust production of extra hair cells in postnatal rat inner ears. Nat Neurosci 2000; 3: 580-586.
8. Raphael Y, Frisancho JC, Roessler BJ. Adenoviral-mediated gene transfer into guinea pig cochlear cells in vivo. Neurosci Lett 1996; 207: 137-141.
9. Derby ML, Sena-Esteves M, Breakefield XO, et al. Gene transfer into the mammalian inner ear using HSV-1 and vaccinia virus vectors. Hear Res 1999; 134: 1-8.
10. Shibata SB, Di Pasquale G, Cortez SR, et al. Gene transfer using bovine adeno-associated virus in the guinea pig cochlea. Gene Ther 2009; 16: 990-997.
11. Sun Y, Tang W, Chang Q, et al. Connexin30 null and conditional connexin26 null mice display distinct pattern and time course of cellular degeneration in the cochlea. J Comp Neurol 2009; 516: 569-579.
12. Wangemann P, Kim HM, Billings S, et al. Developmental delays consistent with cochlear hypothyroidism contribute to failure to develop hearing in mice lacking Slc26a4/pendrin expression. Am J Physiol Renal Physiol 2009; 297: F1435-F1447.
13. Pietola L, Aarnisalo AA, Joensuu J, et al. HOX-GFP and WOX-GFP lentivirus vectors for inner ear gene transfer. Acta Otolaryngol 2008; 128: 613-620.
14. Kho ST, Pettis RM, Mhatre AN, et al. Safety of adeno-associated virus as cochlear gene transfer vector: analysis of distant spread beyond injected cochleae. Mol Ther 2000; 2: 368-373.
15. Ishimoto S, Kawamoto K, Kanzaki S, et al. Gene transfer into supporting cells of the organ of Corti. Hear Res 2002; 173: 187-197.
16. Li Duan M, Bordet T, Mezzina M, et al. Adenoviral and adeno-associated viral vector mediated gene transfer in the guinea pig cochlea. Neuroreport 2002; 13: 1295-1299.
17. Carvalho GJ, Lalwani AK. The effect of cochleostomy and intracochlear infusion on auditory brain stem response threshold in the guinea pig. Am J Otol 1999; 20: 87-90.
18. Duan M, Venail F, Spencer N, et al. Treatment of peripheral sensorineural hearing loss: gene therapy. Gene Ther 2004; 11(Suppl 1): S51-S56.
19. Hildebrand MS, Newton SS, Gubbels SP, et al. Advances in molecular and cellular therapies for hearing loss. Mol Ther 2008; 16: 224-236.
20. Bedrosian JC, Gratton MA, Brigande JV, et al. In vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory function. Mol Ther 2006; 14: 328-335.
21. Aurnhammer C, Haase M, Muether N, et al. Universal real-time PCR for the detection and quantification of adeno-associated virus serotype 2-derived inverted terminal repeat sequences. Hum Gene Ther Methods 2012; 23: 18-28.
22. Thorne M, Salt AN, DeMott JE, et al. Cochlear fluid space dimensions for six species derived from reconstructions of three-dimensional magnetic resonance images. Laryngoscope 1999; 109: 1661-1668.
23. Thompson DC, McPhillips H, Davis RL, et al. Universal newborn hearing screening: summary of evidence. JAMA 2001; 286: 2000-2010.
24. Ahmad S, Tang W, Chang Q, et al. Restoration of connexin26 protein level in the cochlea completely rescues hearing in a mouse model of human connexin30-linked deafness. Proc Natl Acad Sci USA 2007; 104: 1337-1341.
25. Han JJ, Mhatre AN, Wareing M, et al. Transgene expression in the guinea pig cochlea mediated by a lentivirus-derived gene transfer vector. Hum Gene Ther 1999; 10: 1867-1873.
26. Steel KP, Hardisty R. Assessing hearing, vision and balance in mice. In What's Wrong with my Mouse? New Interplays Between Mouse Genes and Behaviour. Society of Neuroscience Short Course Syllabus: Washington, DC, 1996; 26-38.
27. Chang Q, Tang W, Ahmad S, et al. Gap junction mediated intercellular metabolite transfer in the cochlea is compromised in connexin30 null mice. PLoS One 2008; 3: e4088.
28. Lalwani A, Walsh B, Reilly P, et al. Long-term in vivo cochlear transgene expression mediated by recombinant adeno-associated virus. Gene Ther 1998; 5: 277-281.
29. Liu Y, Okada T, Sheykholeslami K, et al. Specific and efficient transduction of cochlear inner hair cells with recombinant adeno-associated virus type 3 vector. Mol Ther 2005; 12: 725-733.
30. Akil O, Seal RP, Burke K, et al. Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy. Neuron 2012; 75: 283-293.
31. Cohen-Salmon M, Ott T, Michel V, et al. Targeted ablation of connexin26 in the inner ear epithelial gap junction network causes hearing impairment and cell death. Curr Biol 2002; 12: 1106-1111.
32. Kikuchi T, Kimura RS, Paul DL, et al. Gap junctions in the rat cochlea: immunohistochemical and ultrastructural analysis. Anat Embryol (Berl) 1995; 191: 101-118.
33. Lalwani AK, Walsh BJ, Reilly PG, et al. Development of in vivo gene therapy for hearing disorders: introduction of adeno-associated virus into the cochlea of the guinea pig. Gene Ther 1996; 3: 588-592.
34. Kilpatrick LA, Li Q, Yang J, et al. Adeno-associated virus-mediated gene delivery into the scala media of the normal and deafened adult mouse ear. Gene Ther 2011; 18: 569-578.
35. Summerford C, Samulski RJ. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998; 72: 1438-1445.
36. Kawamoto K, Oh SH, Kanzaki S, et al. The functional and structural outcome of inner ear gene transfer via the vestibular and cochlear fluids in mice. Mol Ther 2001; 4: 575-585.
37. Stover T, Yagi M, Raphael Y. Cochlear gene transfer: round window vs. cochleostomy inoculation. Hear Res 1999; 136: 124-130.
38. Jero J, Mhatre AN, Tseng CJ, et al. Cochlear gene delivery through an intact round window membrane in mouse. Hum Gene Ther 2001; 12: 539-548.
39. Wang H, Murphy R, Taaffe D, et al. Efficient cochlear gene transfection in guinea-pigs with adeno-associated viral vectors by partial digestion of round window membrane. Gene Ther 2012; 19: 255-263.
40. Iizuka T, Kanzaki S, Mochizuki H, et al. Noninvasive in vivo delivery of transgene via adeno-associated virus into supporting cells of the neonatal mouse cochlea. Hum Gene Ther 2008; 19: 384-390.