Progress Towards Liver and Lung-Directed Gene Therapy with Helper-Dependent Adenoviral Vectors

被引：27
|
作者：
Brunetti-Pierri, Nicola [1 ]
Ng, Philip [1 ]
机构：
[1] Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA
来源：
CURRENT GENE THERAPY | 2009年 / 9卷 / 05期
基金：
美国国家卫生研究院;
关键词：
Gene therapy; adenoviral vectors; helper-dependent adenoviral vectors; gutless; cystic fibrosis; INNATE IMMUNE-RESPONSE; POLYETHYLENE-GLYCOL MODIFICATION; CONDUCTANCE REGULATOR CDNA; KUPFFER CELLS LEADS; IN-VIVO; NONHUMAN-PRIMATES; TRANSGENE EXPRESSION; RECOMBINANT ADENOVIRUSES; CYSTIC-FIBROSIS; MEDIATED TRANSFER;
D O I：
10.2174/156652309789753310
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
Helper-dependent adenoviral vectors (HDAd) have several characteristics making them very attractive for human gene therapy. These vectors are completely devoid of viral coding sequences and are able to mediate high efficiency transduction in vivo to direct high level transgene expression with negligible chronic toxicity. Progress towards liver and lung directed gene therapy with HDAd as well as the current obstacles facing human applications and possible strategies to overcome these obstacles are discussed.
引用
收藏
页码：329 / 340
页数：12
相关论文
共 50 条
  • [1] Progress towards the clinical application of helper-dependent adenoviral vectors for liver and lung gene therapy
    Brunetti-Pierri, Nicola
    Ng, Philip
    [J]. CURRENT OPINION IN MOLECULAR THERAPEUTICS, 2006, 8 (05) : 446 - 454
  • [2] Helper-dependent adenoviral vectors for liver-directed gene therapy
    Brunetti-Pierri, Nicola
    Ng, Philip
    [J]. HUMAN MOLECULAR GENETICS, 2011, 20 : R7 - R13
  • [3] Helper-dependent adenoviral vectors for gene therapy
    Palmer, DJ
    Ng, P
    [J]. HUMAN GENE THERAPY, 2005, 16 (01) : 1 - 16
  • [4] Helper-dependent adenoviral vectors in experimental gene therapy
    Józkowicz, A
    Dulak, J
    [J]. ACTA BIOCHIMICA POLONICA, 2005, 52 (03) : 589 - 599
  • [5] Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors
    Brunetti-Pierri, N.
    Ng, P.
    [J]. GENE THERAPY, 2008, 15 (08) : 553 - 560
  • [6] Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors
    N Brunetti-Pierri
    P Ng
    [J]. Gene Therapy, 2008, 15 : 553 - 560
  • [7] Helper-Dependent Adenoviral Vectors for Liver-Directed Gene Therapy of Primary Hyperoxaluria Type 1
    Castello, Raffaele
    DAria, Stefania
    Borzone, Roberta
    Piccolo, Pasquale
    Annunziata, Patrizia
    Brunetti-Pierri, Nicola
    [J]. MOLECULAR THERAPY, 2013, 21 : S139 - S139
  • [8] Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1
    Castello, R.
    Borzone, R.
    D'Aria, S.
    Annunziata, P.
    Piccolo, P.
    Brunetti-Pierri, N.
    [J]. GENE THERAPY, 2016, 23 (02) : 129 - 134
  • [9] Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1
    R Castello
    R Borzone
    S D'Aria
    P Annunziata
    P Piccolo
    N Brunetti-Pierri
    [J]. Gene Therapy, 2016, 23 : 129 - 134
  • [10] Helper-Dependent Adenoviral Vectors for Liver-Directed Gene Therapy of Primary Hyperoxaluria Type 1
    Castello, Raffaele
    Borzone, Roberta
    Piccolo, Pasquale
    Annunziata, Patrizia
    Brunetti-Pierri, Nicola
    [J]. MOLECULAR THERAPY, 2014, 22 : S227 - S227
12345