Sucess in sight: The eyes have it! Ocular gene therapy trials for LCA look promising

被引：27

作者：

Bainbridge, J. W. ^{[1
]}

Ali, R. R. ^{[1
]}

机构：

[1] UCL, Dept Mol Therapy, Inst Ophthalmol, London EC1V 9EL, England

来源：

GENE THERAPY | 2008年 / 15卷 / 17期

关键词：

D O I：

10.1038/gt.2008.117

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

引用

页码：1191 / 1192

页数：2

共 2 条

[1] Effect of gene therapy on visual function in Leber's congenital amaurosis [J].

Bainbridge, James W. B. ;

Smith, Alexander J. ;

Barker, Susie S. ;

Robbie, Scott ;

Henderson, Robert ;

Balaggan, Kamaljit ;

Viswanathan, Ananth ;

Holder, Graham E. ;

Stockman, Andrew ;

Tyler, Nick ;

Petersen-Jones, Simon ;

Bhattacharya, Shomi S. ;

Thrasher, Adrian J. ;

Fitzke, Fred W. ;

Carter, Barrie J. ;

Rubin, Gary S. ;

Moore, Anthony T. ;

Ali, Robin R. .

NEW ENGLAND JOURNAL OF MEDICINE, 2008, 358 (21) :2231-2239

[2] Safety and efficacy of gene transfer for Leber's congenital amaurosis [J].

Maguire, Albert M. ;

Simonelli, Francesca ;

Pierce, Eric A. ;

Pugh, Edward N., Jr. ;

Mingozzi, Federico ;

Bennicelli, Jeannette ;

Banfi, Sandro ;

Marshall, Kathleen A. ;

Testa, Francesco ;

Surace, Enrico M. ;

Rossi, Settimio ;

Lyubarsky, Arkady ;

Arruda, Valder R. ;

Konkle, Barbara ;

Stone, Edwin ;

Sun, Junwei ;

Jacobs, Jonathan ;

Dell'Osso, Lou ;

Hertle, Richard ;

Ma, Jian-xing ;

Redmond, T. Michael ;

Zhu, Xiaosong ;

Hauck, Bernd ;

Zelenaia, Olga ;

Shindler, Kenneth S. ;

Maguire, Maureen G. ;

Wright, J. Fraser ;

Volpe, Nicholas J. ;

McDonnell, Jennifer Wellman ;

Auricchio, Alberto ;

High, Katherine A. ;

Bennett, Jean .

NEW ENGLAND JOURNAL OF MEDICINE, 2008, 358 (21) :2240-2248

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