Gene Therapy

Gene
Vector
Recipient animal
References
Catalase
AdV
Guinea pig
[32]
Atoh1
AdV
Guinea pig
[4, 35]
Atoh1
AdV
Mouse
[36, 37]
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.
Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science. 1995;270:475–80.PubMedCrossRef
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.
Van de Water TR, Staecker H, Halterman MW, Federoff HJ. Gene therapy in the inner ear. Mechanisms and clinical implications. Ann N Y Acad Sci. 1999;884:345–60.PubMedCrossRef
4.
Izumikawa M, Minoda R, Kawamoto K, Abrashkin KA, Swidershi DL, Dolan DF, et al. Auditory hair cell replacement and hearing improvement by Atoh1 gene therapy in deaf mammals. Nat Med. 2005;11:271–6.PubMedCrossRef
5.
Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 1998;391:806–11.PubMedCrossRef
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.
Maeda Y, Fukushima K, Nishizaki K, Smith RJ. In vitro and in vivo suppression of GJB2 expression by RNA interference. Hum Mol Genet. 2005;14:1641–50.PubMedCrossRef
Mar 26, 2017 | Posted by in OTOLARYNGOLOGY | Comments Off on Gene Therapy

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