Efficient and specific transduction of cochlear supporting cells by adeno-associated virus serotype 5

被引：24

作者：

Ballana, Ester ^{[2
]}

Wang, Jing ^{[3
]}

Venail, Frederic ^{[3
]}

Estivill, Xavier ^{[2
,4
]}

Puel, Jean-Luc ^{[3
]}

Arbones, Maria L. ^{[2
,5
]}

Bosch, Assumpcio ^{[1
]}

机构：

[1] Univ Autonoma Barcelona, CBATEG, Dept Biochem & Mol Biol, E-08193 Barcelona, Spain

[2] UPF, CRG, Genes & Dis Programme, Barcelona, Catalonia, Spain

[3] INSERM, U583, Montpellier, France

[4] Ctr Biomed Res Epidemiol & Publ Hlth CIBERESP, Barcelona, Catalonia, Spain

[5] Ctr Biomed Res Rare Dis CIBERER, Barcelona, Catalonia, Spain

来源：

NEUROSCIENCE LETTERS | 2008年 / 442卷 / 02期

关键词：

gene transfer; AAV5; serotype; inner ear; supporting cells;

D O I：

10.1016/j.neulet.2008.06.060

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

Congenital deafness, affecting 1 in 1000 neonates, can lead to major problems in speech, cognitive and psychosocial development. Congenital deafness is mainly caused by mutations in connexins, hemi-channel proteins forming gap-junctions between supporting cells in the sensory epithelia. We describe a high tropism of AAV5 serotype for the supporting cells of the cochlea, both in vitro in postnatal day 4 mouse explants, and in vivo in the adult guinea-pig inner ear, through scala media perfusion. AAV5 transduction correlates with PDGFR alpha expression, previously reported as AAV5 receptor. This vector could be of major interest in addressing gene therapy approaches to deafness as well as for studying basic aspects of inner-ear development and hearing mechanisms. (c) 2008 Elsevier Ireland Ltd. All rights reserved.

引用

页码：134 / 139

页数：6

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