Gene therapy rescues cilia defects and restores olfactory function in a mammalian ciliopathy model

被引：0

作者：

Jeremy C McIntyre

Erica E Davis

Ariell Joiner

Corey L Williams

I-Chun Tsai

Paul M Jenkins

Dyke P McEwen

Lian Zhang

John Escobado

Sophie Thomas

Katarzyna Szymanska

Colin A Johnson

Philip L Beales

Eric D Green

James C Mullikin

NISC Comparative Sequencing Program

Aniko Sabo

Donna M Muzny

Richard A Gibbs

Tania Attié-Bitach

Bradley K Yoder

Randall R Reed

Nicholas Katsanis

Jeffrey R Martens

机构：

[1] University of Michigan,Department of Pharmacology

[2] Center for Human Disease Modeling,Department of Pediatrics

[3] Duke University Medical Center,Department of Cell Biology

[4] Duke University Medical Center,Département de Génétique et INSERM U

[5] Duke University Medical Center,781

[6] Hôpital Necker-Enfants Malades,Department of Cell Biology

[7] Université Paris Descartes,Department of Molecular Biology and Genetics

[8] Section of Ophthalmology and Neurosciences,undefined

[9] Leeds Institute of Molecular Medicine,undefined

[10] St. James's University Hospital,undefined

[11] Molecular Medicine Unit,undefined

[12] Institute of Child Health,undefined

[13] University College London,undefined

[14] US National Institutes of Health Intramural Sequencing Center,undefined

[15] National Human Genome Research Institute,undefined

[16] National Institutes of Health,undefined

[17] Human Genome Sequencing Center,undefined

[18] Baylor College of Medicine,undefined

[19] University of Alabama at Birmingham,undefined

[20] Johns Hopkins University,undefined

来源：

Nature Medicine | 2012年 / 18卷

关键词：

D O I：

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中图分类号：

学科分类号：

摘要：

Ciliopathies are caused by alterations in the development and function of cilia. Now Jeffrey Martens and his colleagues demonstrate anatomic and functional rescue of cilia development in mature, differentiated neurons by adenovirus-mediated restoration of expression of the wild-type protein intraflagellar transport protein 88 (Ift88) and show restoration of olfactory function in a mouse model of ciliopathy. A loss-of-function mutation in IFT88 is also identified in individuals with ciliopathies.

引用

页码：1423 / 1428

页数：5

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