Predictable Protein Expression with Enhanced Factor IX Activity Following Administration of a Modified AAV5-hFIX Vector to Nonhuman Primates

被引:0
|
作者
Liu, Ying Poi
Lubelski, Jacek
Ehlert, Erich
Gielen, Sander
Miranda, Paula
de Haan, Martin
Nijmeijer, Bart
Petry, Harald
机构
关键词
D O I
暂无
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
2058
引用
收藏
页数:2
相关论文
共 28 条
  • [21] Optimized Adeno-Associated Virus (AAV)-Protein Phosphatase-5 Helper Viruses for Efficient Liver Transduction by Single-Stranded AAV Vectors: Therapeutic Expression of Factor IX at Reduced Vector Doses
    Jayandharan, Giridhara R.
    Zhong, Li
    Sack, Brandon K.
    Rivers, Angela E.
    Li, Mengxin
    Li, Baozheng
    Herzog, Roland W.
    Srivastava, Arun
    HUMAN GENE THERAPY, 2010, 21 (03) : 271 - 283
  • [22] Using Recombinant Adeno-Associated Viral Vectors for Long-Term Expression of a Hyperactive Human Factor IX Mutant in Hemophilic Mice and Comparison of AAV-LK03 and AAV-KP1 in Nonhuman Primates
    Pekrun, Katja
    Stephens, Calvin J.
    Zhang, Feijie
    Kelly, Lauren
    Le Moan, Natacha
    Tarantal, Alice F.
    Blouse, Grant E.
    Kay, Mark A.
    MOLECULAR THERAPY, 2022, 30 (04) : 330 - 330
  • [23] Genetic Modification of the AAV5 Capsid Resulting in De-Targeted Liver and Enhanced Vector Delivery to Lung Following Intravenous Administration
    Stiles, Katie M.
    Frenk, Esther Z.
    Kaminsky, Stephen M.
    Crystal, Ronald G.
    MOLECULAR THERAPY, 2018, 26 (05) : 318 - 318
  • [24] Expression of human FIX at therapeutic levels in nonhuman primates following a single peripheral vein infusion of low doses of self complementary AAV vector renews hope for haemophilia B gene therapy
    Mcintosh, J
    Cochrane, M
    Tuddenham, EGD
    Davidoff, AM
    Nathwani, AC
    BRITISH JOURNAL OF HAEMATOLOGY, 2006, 133 : 18 - 18
  • [25] Expression of Therapeutic Levels of Human Clotting Factor IX in Non Human Primates from an AAV5-Based Gene Therapeutic Vector Generated Using a Fully-Scalable GMP-Compliant Production System
    Nijmeijer, Bart A.
    Petry, Harald
    Sonnemans, Marc A.
    Salmon, Florence
    Preusting, Hans
    Van Oorschot, Arie
    Twisk, Jaap
    MOLECULAR THERAPY, 2012, 20 : S226 - S226
  • [26] A murine FVIIa variant with increased tissue factor-independent intrinsic activity corrects the murine hemophilia B phenotype following AAV administration: Implications for therapeutic AAV vector doses.
    Margaritis, Paris
    Roy, Elise
    High, Katherine A.
    BLOOD, 2006, 108 (11) : 935A - 935A
  • [27] Liver targeted delivery of recombinant adeno-associated virus (rAAV) serotype 5 vectors leads to sustained therapeutic expression of human factor IX (hFIX) in non-human primates previously sensitized to rAAV2 vectors.
    Nathwani, AC
    Ng, CY
    Zhou, JF
    Spence, Y
    Nienhuis, AW
    Davidoff, AM
    BLOOD, 2003, 102 (11) : 493A - 493A
  • [28] Sustained factor IX activity levels and bleeding protection following etranacogene dezaparvovec administration in people with haemophilia B without and with adeno-associated virus serotype 5 neutralizing antibodies
    Miesbach, W.
    Leebeek, F. W. G.
    Recht, M.
    Key, N. S.
    Lattimore, S.
    Castaman, G.
    Cooper, D. L.
    Verweij, S.
    Dolmetsch, R.
    Tarrant, J.
    Li, Y.
    Monahan, P. E.
    Pipe, S. W.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A4 - A5