Birth of a New Therapeutic Platform: 47 Years of Adeno-associated Virus Biology From Virus Discovery to Licensed Gene Therapy

被引：16

作者：

Flotte, Terence R. ^{[1
]}

机构：

[1] Univ Massachusetts, Sch Med, Dept Pediat, Gene Therapy Ctr, Worcester, MA USA

来源：

MOLECULAR THERAPY | 2013年 / 21卷 / 11期

关键词：

ADENO-ASSOCIATED VIRUS; LEBER CONGENITAL AMAUROSIS; PHASE-I TRIAL; TRANSMEMBRANE CONDUCTANCE REGULATOR; CYSTIC-FIBROSIS; MESSENGER-RNA; CLINICAL-TRIAL; WILD-TYPE; INTRAMUSCULAR INJECTION; RECOMBINANT PLASMID;

D O I：

10.1038/mt.2013.226

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

引用

页码：1976 / 1981

页数：6

共 99 条

[61]

KYOSTIO SRM, 1994, J VIROL, V68, P2947

[62] CLONING OF INFECTIOUS ADENO-ASSOCIATED VIRUS GENOMES IN BACTERIAL PLASMIDS [J].

LAUGHLIN, CA ;

TRATSCHIN, JD ;

COON, H ;

CARTER, BJ .

GENE, 1983, 23 (01) :65-73

[63] Long-Term Follow-Up After Gene Therapy for Canavan Disease [J].

Leone, Paola ;

Shera, David ;

McPhee, Scott W. J. ;

Francis, Jeremy S. ;

Kolodny, Edwin H. ;

Bilaniuk, Larissa T. ;

Wang, Dah-Jyuu ;

Assadi, Mitra ;

Goldfarb, Olga ;

Goldman, H. Warren ;

Freese, Andrew ;

Young, Deborah ;

During, Matthew J. ;

Samulski, R. Jude ;

Janson, Christopher G. .

SCIENCE TRANSLATIONAL MEDICINE, 2012, 4 (165)

[64] Characterization of the adenoassociated virus Rep protein complex formed on the viral origin of DNA replication [J].

Li, Z ;

Brister, JR ;

Im, DS ;

Muzyczka, N .

VIROLOGY, 2003, 313 (02) :364-376

[65] 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

[66] Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial [J].

Maguire, Albert M. ;

High, Katherine A. ;

Auricchio, Alberta ;

Wright, J. Fraser ;

Pierce, Eric A. ;

Testa, Francesco ;

Mingozzi, Federico ;

Bennicelli, Jeannette L. ;

Ying, Gui-shuang ;

Rossi, Settimio ;

Fulton, Ann ;

Marshall, Kathleen A. ;

Banfi, Sandra ;

Chung, Daniel C. ;

Morgan, Jessica I. W. ;

Hauck, Bernd ;

Zelenaia, Olga ;

Zhu, Xiaosong ;

Raffini, Leslie ;

Coppieters, Frauke ;

De Baere, Elfride ;

Shindler, Kenneth S. ;

Volpe, Nicholas J. ;

Surace, Enrico M. ;

Acerra, Carmela ;

Lyubarsky, Arkady ;

Redmond, T. Michael ;

Stone, Edwin ;

Sun, Junwei ;

McDonnell, Jennifer Wellman ;

Leroy, Bart P. ;

Simonelli, Francesca ;

Bennett, Jean .

LANCET, 2009, 374 (9701) :1597-1605

[67] AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B [J].

Manno, CS ;

Chew, AJ ;

Hutchison, S ;

Larson, PJ ;

Herzog, RW ;

Arruda, VP ;

Tai, SJ ;

Ragni, MV ;

Thompson, A ;

Ozelo, M ;

Couto, LB ;

Leonard, DGB ;

Johnson, FA ;

McClelland, A ;

Scallan, C ;

Skarsgard, E ;

Flake, AW ;

Kay, MA ;

High, KA ;

Glader, B .

BLOOD, 2003, 101 (08) :2963-2972

[68] Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis - A multicenter, double-blind, placebo-controlled trial [J].

Moss, RB ;

Rochman, D ;

Spencer, LT ;

Aitken, ML ;

Zeitlhi, PL ;

Waltz, D ;

Milla, C ;

Brody, AS ;

Clancy, JP ;

Ramsey, B ;

Hamblett, N ;

Heald, AE .

CHEST, 2004, 125 (02) :509-521

[69] A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease [J].

Muramatsu, Shin-ichi ;

Fujimoto, Ken-ichi ;

Kato, Seiya ;

Mizukami, Hiroaki ;

Asari, Sayaka ;

Ikeguchi, Kunihiko ;

Kawakami, Tadataka ;

Urabe, Masashi ;

Kume, Akihiro ;

Sato, Toshihiko ;

Watanabe, Eiju ;

Ozawa, Keiya ;

Nakano, Imaharu .

MOLECULAR THERAPY, 2010, 18 (09) :1731-1735

[70] Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B [J].

Nathwani, Amit C. ;

Tuddenham, Edward G. D. ;

Rangarajan, Savita ;

Rosales, Cecilia ;

McIntosh, Jenny ;

Linch, David C. ;

Chowdary, Pratima ;

Riddell, Anne ;

Pie, Arnulfo Jaquilmac ;

Harrington, Chris ;

O'Beirne, James ;

Smith, Keith ;

Pasi, John ;

Glader, Bertil ;

Rustagi, Pradip ;

Ng, Catherine Y. C. ;

Kay, Mark A. ;

Zhou, Junfang ;

Spence, Yunyu ;

Morton, Christopher L. ;

Allay, James ;

Coleman, John ;

Sleep, Susan ;

Cunningham, John M. ;

Srivastava, Deokumar ;

Basner-Tschakarjan, Etiena ;

Mingozzi, Federico ;

High, Katherine A. ;

Gray, John T. ;

Reiss, Ulrike M. ;

Nienhuis, Arthur W. ;

Davidoff, Andrew M. .

NEW ENGLAND JOURNAL OF MEDICINE, 2011, 365 (25) :2357-2365

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