Toward gene therapy for hemophilia A with novel adenoviral vectors: successes and limitations in canine models

被引:12
作者
Arruda, VR [1 ]
机构
[1] Univ Penn, Childrens Hosp Philadelphia, Sch Med, Dept Pediat, Philadelphia, PA 19104 USA
关键词
D O I
10.1111/j.1538-7836.2006.01964.x
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:1215 / 1217
页数:3
相关论文
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  • [1] FACTOR-VIII GENE INVERSIONS IN SEVERE HEMOPHILIA-A - RESULTS OF AN INTERNATIONAL CONSORTIUM STUDY
    ANTONARAKIS, SE
    ROSSITER, JP
    YOUNG, M
    HORST, J
    DEMOERLOOSE, P
    SOMMER, SS
    KETTERLING, RP
    KAZAZIAN, HH
    NEGRIER, C
    VINCIGUERRA, C
    GITSCHIER, J
    GOOSSENS, M
    GIRODON, E
    GHANEM, N
    PLASSA, F
    LAVERGNE, JM
    VIDAUD, M
    COSTA, JM
    LAURIAN, Y
    LIN, SW
    LIN, SR
    SHEN, MC
    LILLICRAP, D
    TAYLOR, SAM
    WINDSOR, S
    VALLEIX, SV
    NAFA, K
    SULTAN, Y
    DELPECH, M
    VNENCAKJONES, CL
    PHILLIPS, JA
    LJUNG, RCR
    KOUMBARELIS, E
    GIALERAKI, A
    MANDALAKI, T
    JENKINS, PV
    COLLINS, PW
    PASI, KJ
    GOODEVE, A
    PEAKE, I
    PRESTON, FE
    SCHWARTZ, M
    SCHEIBEL, E
    INGERSLEV, J
    COOPER, DN
    MILLAR, DS
    KAKKAR, VV
    GIANNELLI, F
    NAYLOR, JA
    TIZZANO, EF
    [J]. BLOOD, 1995, 86 (06) : 2206 - 2212
  • [2] Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A
    Brown, BD
    Shi, CX
    Powell, S
    Hurlbut, D
    Graham, FL
    Lillicrap, D
    [J]. BLOOD, 2004, 103 (03) : 804 - 810
  • [3] Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
    Chuah, MKL
    Schiedner, G
    Thorrez, L
    Brown, B
    Johnston, M
    Gillijns, V
    Hertel, S
    Van Rooijen, N
    Lillicrap, D
    Collen, D
    VandenDriessche, T
    Kochanek, S
    [J]. BLOOD, 2003, 101 (05) : 1734 - 1743
  • [4] A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia
    Ehrhardt, A
    Xu, H
    Dillow, AM
    Bellinger, DA
    Nichols, TC
    Kay, MA
    [J]. BLOOD, 2003, 102 (07) : 2403 - 2411
  • [5] Germline alteration by gene therapy: assessing and reducing the risks
    Gordon, JW
    [J]. MOLECULAR MEDICINE TODAY, 1998, 4 (11): : 468 - 470
  • [6] Gene therapy for hemophilia: an imperative to succeed
    Hough, C
    Lillicrap, D
    [J]. JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2005, 3 (06) : 1195 - 1205
  • [7] Aberrant splicing and premature termination of transcription of the FVIII gene as a cause of severe canine hemophilia A: Similarities with the intron 22 inversion mutation in human hemophilia
    Hough, C
    Kamisue, S
    Cameron, C
    Notley, C
    Tinlin, S
    Giles, A
    Lillicrap, D
    [J]. THROMBOSIS AND HAEMOSTASIS, 2002, 87 (04) : 659 - 665
  • [8] Factor VIII alloantibodies in hemophilia
    Jacquemin, MG
    Saint-Remy, JMR
    [J]. CURRENT OPINION IN HEMATOLOGY, 2004, 11 (03) : 146 - 150
  • [9] The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion
    Lozier, JN
    Dutra, A
    Pak, E
    Zhou, N
    Zheng, ZL
    Nichols, TC
    Bellinger, DA
    Read, M
    Morgan, RA
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2002, 99 (20) : 12991 - 12996
  • [10] Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model
    McCormack, W. M.
    Seiler, M. P.
    Bertin, T. K.
    Ubhayakar, K.
    Palmer, D. J. .
    Ng, P.
    Nichols, T. C.
    Lee, B.
    [J]. JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2006, 4 (06) : 1218 - 1225