Revisiting the "New'' Inflammatory Toxicities of Adeno-Associated Virus Vectors

被引：33

作者：

Flotte, Terence R.

机构：

来源：

HUMAN GENE THERAPY | 2020年 / 31卷 / 7-8期

关键词：

CYSTIC-FIBROSIS; TOXICOLOGY; RESPONSES; DELIVERY; SAFETY;

D O I：

10.1089/hum.2020.29117.trf

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：398 / 399

页数：2

共 12 条

[1] Repeated delivery of adeno-associated virus vectors to the rabbit airway [J].

Beck, SE ;

Jones, LA ;

Chesnut, K ;

Walsh, SM ;

Reynolds, TC ;

Carter, BJ ;

Askin, FB ;

Flotte, TR ;

Guggino, WB .

JOURNAL OF VIROLOGY, 1999, 73 (11) :9446-9455

[2]

Conrad CK, 1996, GENE THER, V3, P658

[3] Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease [J].

Corti, Manuela ;

Liberati, Cristina ;

Smith, Barbara K. ;

Lawson, Lee Ann ;

Tuna, Ibrahim S. ;

Conlon, Thomas J. ;

Coleman, Kirsten E. ;

Islam, Saleem ;

Herzog, Roland W. ;

Fuller, David D. ;

Collins, Shelley W. ;

Byrne, Barry J. .

HUMAN GENE THERAPY CLINICAL DEVELOPMENT, 2017, 28 (04) :208-218

[4] STABLE IN-VIVO EXPRESSION OF THE CYSTIC-FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR WITH AN ADENOASSOCIATED VIRUS VECTOR [J].

FLOTTE, TR ;

AFIONE, SA ;

CONRAD, C ;

MCGRATH, SA ;

SOLOW, R ;

OKA, H ;

ZEITLIN, PL ;

GUGGINO, WB ;

CARTER, BJ .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1993, 90 (22) :10613-10617

[5] Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN [J].

Hinderer, Christian ;

Katz, Nathan ;

Buza, Elizabeth L. ;

Dyer, Cecilia ;

Goode, Tamara ;

Bell, Peter ;

Richman, Laura K. ;

Wilson, James M. .

HUMAN GENE THERAPY, 2018, 29 (03) :285-298

[6] Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques [J].

Hordeaux, Juliette ;

Hinderer, Christian ;

Goode, Tamara ;

Buza, Elizabeth L. ;

Bell, Peter ;

Calcedo, Roberto ;

Richman, Laura K. ;

Wilson, James M. .

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2018, 10 :68-78

[7] Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques [J].

Hordeaux, Juliette ;

Hinderer, Christian ;

Goode, Tamara ;

Katz, Nathan ;

Buza, Elizabeth L. ;

Bell, Peter ;

Calcedo, Roberto ;

Richman, Laura K. ;

Wilson, James M. .

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2018, 10 :79-88

[8] CD8+ T-cell responses to adeno-associated virus capsid in humans [J].

Mingozzi, Federico ;

Maus, Marcela V. ;

Hui, Daniel J. ;

Sabatino, Denise E. ;

Murphy, Samuel L. ;

Rasko, John E. J. ;

Ragni, Margaret V. ;

Manno, Catherine S. ;

Sommer, Jurg ;

Jiang, Haiyan ;

Pierce, Glenn F. ;

Ertl, Hildegund C. J. ;

High, Katherine A. .

NATURE MEDICINE, 2007, 13 (04) :419-422

[9] Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: A Randomized placebo-controlled phase 2B trial [J].

Moss, Richard B. ;

Milla, Carlos ;

Colombo, John ;

Accurso, Frank ;

Zeitlin, Pamela L. ;

Clancy, John P. ;

Spencer, L. Terry ;

Pilewski, Joseph ;

Waltz, David A. ;

Dorkin, Henry L. ;

Ferkol, Thomas ;

Pian, Mark ;

Ramsey, Bonnie ;

Carter, Barrie J. ;

Martin, Dana B. ;

Heald, Alison E. .

HUMAN GENE THERAPY, 2007, 18 (08) :726-732

[10] Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression [J].

Mueller, Christian ;

Chulay, Jeffrey D. ;

Trapnell, Bruce C. ;

Humphries, Margaret ;

Carey, Brenna ;

Sandhaus, Robert A. ;

McElvaney, Noel G. ;

Messina, Louis ;

Tang, Qiushi ;

Rouhani, Farshid N. ;

Campbell-Thompson, Martha ;

Fu, Ann Dongtao ;

Yachnis, Anthony ;

Knop, David R. ;

Ye, Guo-jie ;

Brantly, Mark ;

Calcedo, Roberto ;

Somanathan, Suryanarayan ;

Richman, Lee P. ;

Vonderheide, Robert H. ;

Hulme, Maigan A. ;

Brusko, Todd M. ;

Wilson, James M. ;

Flotte, Terence R. .

JOURNAL OF CLINICAL INVESTIGATION, 2013, 123 (12) :5310-5318

← 1 2 →