Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

被引：168

作者：

Cideciyan, Artur V. ^{[1
]}

Hauswirth, William W. ^{[2
]}

Aleman, Tomas S. ^{[1
]}

Kaushal, Shalesh ^{[2
]}

Schwartz, Sharon B. ^{[1
]}

Boye, Sanford L. ^{[2
]}

Windsor, Elizabeth A. M. ^{[1
]}

Conlon, Thomas J. ^{[2
]}

Sumaroka, Alexander ^{[1
]}

Roman, Alejandro J. ^{[1
]}

Byrne, Barry J. ^{[2
]}

Jacobson, Samuel G. ^{[1
]}

机构：

[1] Univ Penn, Philadelphia, PA 19104 USA

[2] Univ Florida, Gainesville, FL USA

来源：

NEW ENGLAND JOURNAL OF MEDICINE | 2009年 / 361卷 / 07期

关键词：

D O I：

10.1056/NEJMc0903652

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：725 / 727

页数：4

共 5 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] Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics [J].

Cideciyan, Artur V. ;

Aleman, Tomas S. ;

Boye, Sanford L. ;

Schwartz, Sharon B. ;

Kaushal, Shalesh ;

Roman, Alejandro J. ;

Pang, Ji-Jing ;

Sumaroka, Alexander ;

Windsor, Elizabeth A. M. ;

Wilson, James M. ;

Flotte, Terence R. ;

Fishman, Gerald A. ;

Heon, Elise ;

Stone, Edwin M. ;

Byrne, Barry J. ;

Jacobson, Samuel G. ;

Hauswirth, William W. .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2008, 105 (39) :15112-15117

[3] Leber congenital amaurosis: Genes, proteins and disease mechanisms [J].

den Hollander, Anneke I. ;

Roepman, Ronald ;

Koenekoop, Robert K. ;

Cremers, Frans P. M. .

PROGRESS IN RETINAL AND EYE RESEARCH, 2008, 27 (04) :391-419

[4] Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial [J].

Hauswirth, William W. ;

Aleman, Tomas S. ;

Kaushal, Shalesh ;

Cideciyan, Artur V. ;

Schwartz, Sharon B. ;

Wang, Lili ;

Conlon, Thomas J. ;

Boye, Sanford L. ;

Flotte, Terence R. ;

Byrne, Barry J. ;

Jacobson, Samuel G. .

HUMAN GENE THERAPY, 2008, 19 (10) :979-990

[5] 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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