Intramuscular delivery of AAV1 vectors resulted in sustained expression of therapeutic levels of FIX in hemophilia B dogs.

被引：0
|
作者：
Chao, HJ
Monahan, PE
Elia, JR
Camp, AS
Nichols, TC
Bellinger, DA
Samulski, RJ
Walsh, CE
机构：
[1] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27515 USA
[2] Univ N Carolina, Dept Pathol, Chapel Hill, NC USA
来源：
BLOOD | 2002年 / 100卷 / 11期
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：
60
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收藏
页码：19A / 20A
页数：2
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  • [1] Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors
    Chao, HJ
    Monahan, PE
    Liu, YB
    Samulski, RJ
    Walsh, CE
    [J]. MOLECULAR THERAPY, 2001, 4 (03) : 217 - 222
  • [2] Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors.
    Chao, HJ
    Monahan, PE
    Liu, YB
    Samulski, RJ
    Walsh, CE
    [J]. BLOOD, 2001, 98 (11) : 704A - 704A
  • [3] Efficient Induction of Tolerance To FIX by Direct Intramuscular Delivery of AAV1
    Cohn, Ellen F.
    Zhuo, Jiacai
    Kelly, Meagan
    Zhang, Jiayin
    Chao, Hengjun
    [J]. MOLECULAR THERAPY, 2006, 13 : S379 - S379
  • [4] Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
    PE Monahan
    RJ Samulski
    J Tazelaar
    X Xiao
    TC Nichols
    DA Bellinger
    MS Read
    CE Walsh
    [J]. Gene Therapy, 1998, 5 : 40 - 49
  • [5] Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
    Monahan, PE
    Samulski, RJ
    Tazelaar, J
    Xiao, X
    Nichols, TC
    Bellinger, DA
    Read, MS
    Walsh, CE
    [J]. GENE THERAPY, 1998, 5 (01) : 40 - 49
  • [6] Subcutaneous recombinant factor IX (beneFIX(TM)) administration produces therapeutic levels of FIX activity in hemophilia B dogs.
    Brinkhous, KM
    Sigman, JL
    Read, MS
    Stewart, PF
    Bush, L
    Bartlett, E
    Rup, B
    Keith, JC
    Garzone, P
    McCarthy, K
    Schaub, RG
    [J]. BLOOD, 1996, 88 (10) : 1757 - 1757
  • [7] Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
    Wang, LL
    Nichols, TC
    Read, MS
    Bellinger, DA
    Verma, IM
    [J]. MOLECULAR THERAPY, 2000, 1 (02) : 154 - 158
  • [8] AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
    Crudele, Julie M.
    Finn, Jonathan D.
    Siner, Joshua I.
    Martin, Nicholas B.
    Niemeyer, Glenn P.
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    Mingozzi, Federico
    Lothrop, Clinton D., Jr.
    Arruda, Valder R.
    [J]. BLOOD, 2015, 125 (10) : 1553 - 1561
  • [9] Characterization of the immune response to canine factor IX following AAV-mediated intravascular gene delivery to skeletal muscle in hemophilia B dogs.
    Hui, DJ
    Mingozzi, F
    Sabatino, DE
    Mccorquodale, S
    Dillow, A
    Nichols, TC
    Arruda, VR
    High, KA
    [J]. BLOOD, 2005, 106 (11) : 377A - 377A
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    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.
    [J]. HAEMATOLOGICA, 2016, 101 : 309 - 309
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