Improved efficacy in gene therapy of hemophilia A using an AAV6 pseudo-typed FVIII vector

被引:0
作者
Jiang, HY [1 ]
Patarroyo-White, S [1 ]
Nagy, D [1 ]
Vargas, J [1 ]
Scallan, C [1 ]
Liu, TY [1 ]
Powell, S [1 ]
Wright, F [1 ]
Couto, L [1 ]
机构
[1] Avigen Inc, Alameda, CA USA
关键词
D O I
暂无
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
471
引用
收藏
页码:S184 / S184
页数:1
相关论文
共 50 条
[41]   Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors [J].
Sabatino, Denise E. ;
Lange, Amy M. ;
Altynova, Ekaterina S. ;
Sarkar, Rita ;
Zhou, Shangzhen ;
Merricks, Elizabeth P. ;
Franck, Helen G. ;
Nichols, Timothy C. ;
Arruda, Valder R. ;
Kazazian, Haig H., Jr. .
MOLECULAR THERAPY, 2011, 19 (03) :442-449
[42]   Reduction of Inhibitory Anti-FVIII Antibody Titer by Using a B Domain Variant FVIII/N6 cDNA for Nonviral Gene Therapy in Hemophilia a Mice [J].
Jirovska, Dominika ;
Ye, Pciqing ;
Pipe, Steven W. ;
Miao, Carol H. .
BLOOD, 2008, 112 (11) :1212-1212
[43]   Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials [J].
Liching Cao ;
Annemarie Ledeboer ;
Yonghua Pan ;
Yanmei Lu ;
Kathleen Meyer .
Gene Therapy, 2023, 30 :150-159
[44]   Long term dose-dependent correction of hemophilia a dogs using AAV-8 and AAV-9-Mediated FVIII gene transfer. [J].
Sabatino, Denise E. ;
Lange, Amy M. ;
Mucci, Melinda ;
Sarkar, Rita ;
Dillow, Aaron M. ;
Nichols, Timothy C. ;
Arruda, Valder R. ;
Kazazian, Haig H. .
BLOOD, 2006, 108 (11) :298A-298A
[45]   Safety and Efficacy of Liver-Directed AAV Gene Therapy in Privately Owned Dogs with Severe Hemophilia A [J].
Van Gorder, Lucas ;
Doshi, Bhavya S. ;
Willis, Elinor ;
Nichols, Timothy C. ;
Lanza, Matthew ;
Cook, Emma ;
Everett, John ;
Merricks, Elizabeth ;
Arruda, Valder R. ;
Bushman, Frederic ;
Samelson-Jones, Benjamin ;
Callan, Mary Beth .
MOLECULAR THERAPY, 2023, 31 (04) :315-316
[46]   Efficacy and Safety of Viral Vector Based Gene Therapy in Patients with Hemophilia a: A Systematic Review [J].
Aiman, Wajeeha ;
Kazmi, Syeda Hafsa ;
Saeed, Memoona ;
Khan, Muhammad Imran ;
Omar, Zainab ;
Suleman, Muhammad ;
Jamil, Taimoor ;
Bhatti, Mahak Khadija ;
Butt, Sigmone Khalid ;
Zubair, Hina ;
Supraja, A. ;
Zafar, Maha ;
Arooj, Yusra ;
Ali, Muhammad Ashar .
BLOOD, 2022, 140 :12992-12993
[47]   Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B [J].
Geoffrey L Rogers ;
Ashley T Martino ;
Irene Zolotukhin ;
Hildegund CJ Ertl ;
Roland W Herzog .
Journal of Translational Medicine, 12
[48]   AAV-MEDIATED GENE THERAPY FOR HEMOPHILIA B-EXPRESSION AT THERAPEUTIC LEVELS WITH LOW VECTOR DOSES [J].
High, K. A. ;
George, L. ;
Sullivan, S. ;
Luk, A. ;
Urich, T. ;
Teitel, J. ;
Cuker, A. ;
Wright, F. ;
Chen, Y. ;
Hui, D. ;
Wachtel, K. ;
Galvao, A. ;
McMillan, L. E. ;
Takefman, D. ;
Carr, M. E. ;
Couto, L. ;
Anguela, X. .
HAEMATOLOGICA, 2016, 101 :309-309
[49]   Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B [J].
Rogers, Geoffrey L. ;
Martino, Ashley T. ;
Zolotukhin, Irene ;
Ertl, Hildegund C. J. ;
Herzog, Roland W. .
JOURNAL OF TRANSLATIONAL MEDICINE, 2014, 12
[50]   Suicide Gene Therapy with Exosome Associated AAV6 Vectors Loaded with an Inducible Caspase 9 Gene is Therapeutic in Hepatocellular Carcinoma Models In Vivo [J].
Khan, Nusrat ;
Bammidi, Sridhar ;
Jayandharan, Giridhara R. .
MOLECULAR THERAPY, 2020, 28 (04) :477-477