Gene
Vector
Recipient animal
References
Catalase
AdV
Guinea pig
[32]
Atoh1
AdV
Guinea pig
Atoh1
AdV
Mouse
GDNF/TGFbeta
AdV
Guinea pig
[31]
NOX3
(siRNA)
Rat
[34]
XIAP
AAV
Rat
[33]
Connexin 26
Liposome (siRNA)
Mouse
[9]
Connexin 26
BAAV
Mouse
[38]
Connexin 30
Electroporation (shRNA)
Mouse
[8]
Ush1c
ASO
Mouse
[7]
VGLUT3
AAV
Mouse
[39]
References
1.
2.
Sokolic R, Kesserwan C, Candotti F. Recent advances in gene therapy for severe congenital immunodeficiency diseases. Curr Opin Hematol. 2008;15:375–80. doi:10.1097/MOH.0b013e328302c807.PubMedCentralPubMedCrossRef
3.
4.
5.
6.
Kole R, Krainer AR, Altman S. RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov. 2012;11:125–40. doi:10.1038/nrd3625.PubMed
7.
Lentz JJ, Jodelka FM, Hinrich AJ, McCaffrey KE, Farris HE, Spalitta MJ, et al. Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness. Nat Med. 2013;19:345–50. doi:10.1038/nm.3106.PubMedCentralPubMedCrossRef
8.
Miwa T, Minoda R, Ise M, Yamada T, Yumoto E. Mouse otocyst transuterine gene transfer restores hearing in mice with connexin 30 deletion-associated hearing loss. Mol Ther. 2013;21:1142–50. doi:10.1038/mt.2013.62.PubMedCentralPubMedCrossRef
9.
10.
Fukui H, Raphael Y. Gene therapy for the inner ear. Hear Res. 2013;297:99–105. doi:10.1016/j.heares.2012.11.017.PubMedCentralPubMedCrossRef