Adeno-associated virus-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy

被引:25
作者
Herzog, RW
High, KA
机构
[1] Childrens Hosp Philadelphia, Div Hematol, Abramson Res Ctr 310A, Philadelphia, PA 19104 USA
[2] Univ Penn, Med Ctr, Dept Pediat, Philadelphia, PA 19104 USA
[3] Univ Penn, Med Ctr, Dept Pathol, Philadelphia, PA 19104 USA
关键词
D O I
10.1055/s-0037-1615877
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:540 / 546
页数:7
相关论文
共 48 条
[41]   Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors [J].
Tripathy, SK ;
Black, HB ;
Goldwasser, E ;
Leiden, JM .
NATURE MEDICINE, 1996, 2 (05) :545-550
[42]   Gene therapy - promises, problems and prospects [J].
Verma, IM ;
Somia, N .
NATURE, 1997, 389 (6648) :239-242
[43]   A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis [J].
Wagner, JA ;
Moran, ML ;
Messner, AH ;
Daifuku, R ;
Conrad, CK ;
Reynolds, T ;
Guggino, WB ;
Moss, RB ;
Carter, BJ ;
Wine, JJ ;
Flotte, TR ;
Gardner, P .
HUMAN GENE THERAPY, 1998, 9 (06) :889-909
[44]   ADENOASSOCIATED VIRUS (AAV) REP PROTEINS MEDIATE COMPLEX-FORMATION BETWEEN AAV DNA AND ITS INTEGRATION SITE IN HUMAN DNA [J].
WEITZMAN, MD ;
KYOSTIO, SRM ;
KOTIN, RM ;
OWENS, RA .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1994, 91 (13) :5808-5812
[45]   Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus [J].
Xiao, X ;
Li, J ;
Samulski, RJ .
JOURNAL OF VIROLOGY, 1998, 72 (03) :2224-2232
[46]   Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector [J].
Xiao, XA ;
Li, JA ;
Samulski, RJ .
JOURNAL OF VIROLOGY, 1996, 70 (11) :8098-8108
[47]   Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro [J].
Yang, CC ;
Xiao, X ;
Zhu, X ;
Ansardi, DC ;
Epstein, ND ;
Frey, MR ;
Matera, AG ;
Samulski, RJ .
JOURNAL OF VIROLOGY, 1997, 71 (12) :9231-9247
[48]  
YAO SN, 1994, GENE THER, V1, P99