Permanent phenotypic correction of Haemophilia B in immunocompetent mice by prenatal gene therapy

被引：0
|
作者：
Waddington, SN
Nivsarkar, M
Mistry, A
Buckley, SMK
Kemball-Cook, G
Mosley, KL
Mitrophanous, K
Radcliffe, P
Holder, M
Brittan, M
Georgiadis, T
Al-Allaf, F
Bigger, B
Gregory, L
Cook, HT
Ali, RR
Thrasher, A
Tuddenham, EGD
Themis, M
Coutelle, C
机构：
[1] Univ London Imperial Coll Sci Technol & Med, Gene Therapy Res Grp, Sect Cell & Mol Biol, London SW7 2AZ, England
[2] Inst Child Hlth, Mol Immunol Unit, London WC1N 1EH, England
[3] Univ London Imperial Coll Sci Technol & Med, MRC, Ctr Clin Sci, London W12 0NN, England
[4] Univ London Imperial Coll Sci Technol & Med, Fac Med, Renal Med Sect, London W12 0NN, England
[5] Oxford BioMed UK Ltd, Oxford OX4 4GA, England
[6] Canc Res UK, Histopathol Unit, London WC2A 3PX, England
[7] UCL, Inst Ophthalmol, Dept Mol Genet, London EC1V 9EL, England
来源：
JOURNAL OF GENE MEDICINE | 2004年 / 6卷 / 09期
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：
42
引用
收藏
页码：S17 / S17
页数：1
相关论文
共 50 条
  • [31] Therapeutic plasma concentrations of human factor IX in mice after gene delivery into the amniotic cavity: A model for the prenatal treatment of haemophilia B
    Schneider, H
    Adebakin, S
    Themis, M
    Cook, T
    Douar, AM
    Pavirani, A
    Coutelle, C
    JOURNAL OF GENE MEDICINE, 1999, 1 (06): : 424 - 432
  • [32] Promoter less gene targeting without nucleases ameliorates haemophilia B in mice
    Barzel, A.
    Paulk, N. K.
    Shi, Y.
    Huang, Y.
    Chu, K.
    Zhang, F.
    Valdmanis, P. N.
    Spector, L. P.
    Porteus, M. H.
    Gaensler, K. M.
    Kay, M. A.
    NATURE, 2015, 517 (7534) : 360 - U476
  • [33] Permanent correction of dystrophic EB by gene therapy in a dog model of the condition
    Pin, D.
    Carozzo, C.
    Meneguzzi, G.
    Gache, Y.
    JOURNAL OF INVESTIGATIVE DERMATOLOGY, 2007, 127 (10) : 2479 - 2479
  • [34] Phenotypic Reversion of Fair Hair upon Gene Therapy of the Phenylketonuria Mice
    Thoeny, Beat
    Ding, Zhaobing
    Rebuffat, Alexandre
    Viecelli, Hiu Man
    HUMAN GENE THERAPY, 2014, 25 (07) : 573 - 574
  • [35] FDA approves $3.5m gene therapy for adults with haemophilia B
    Tanne, Janice Hopkins
    BMJ-BRITISH MEDICAL JOURNAL, 2022, 379 : o2858
  • [36] Defect on X-Chromosome Gene Therapy shows Success in Haemophilia B
    Ruchalla, Elke
    TRANSFUSIONSMEDIZIN, 2012, 2 (02) : 76 - 76
  • [37] Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
    Mount, JD
    Herzog, RW
    Tillson, DM
    Goodman, SA
    Robinson, N
    McCleland, ML
    Bellinger, D
    Nichols, TC
    Arruda, VR
    Lothrop, CD
    High, KA
    BLOOD, 2002, 99 (08) : 2670 - 2676
  • [38] FACING THE BLEEDING OBVIOUS: AAV-MEDIATED GENE THERAPY FOR HAEMOPHILIA B
    Rasko, J. E. J.
    High, Katherine
    Tigges, Michael
    Manno, Catherine
    Sabatino, Denise
    Dake, Michael
    McDonnell, Jennifer Wellman
    Razavi, Mahmood
    Arruda, Valder
    Herzog, Roland
    Rustagi, Pradip
    Sommer, Jurg
    Ragni, Margaret
    Konkle, Barbara
    Lessard, Ruth
    Luk, Alvin
    Glader, Bertil
    Pierce, Glenn
    Couto, Linda
    Jiang, Haiyan
    Kay, Mark
    JOURNAL OF GENE MEDICINE, 2009, 11 (09): : 843 - 843
  • [39] In Vivo Enrichment of Genetically Manipulated Platelets for Murine Haemophilia B Gene Therapy
    Chen, Yingyu
    Schroeder, Jocelyn A.
    Hu, Jianda
    Shi, Qizhen
    BLOOD, 2018, 132
  • [40] CONCEPTUAL FRAMEWORK FOR EVALUATING THE COST-EFFECTIVENESS OF A GENE THERAPY FOR HAEMOPHILIA B
    Li, N.
    Sawyer, E. K.
    Maruszczyk, K.
    Slomka, M.
    Burke, T.
    Martin, A.
    O'Hara, J.
    VALUE IN HEALTH, 2019, 22 : S847 - S848
12345