Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis

被引:48
|
作者
Flotte, Terence R.
Ng, Philip
Dylla, Doug E.
McCray, Paul B., Jr.
Wang, Guoshun
Kolls, Jay K.
Hu, Jim
机构
[1] Univ Toronto, Hosp Sick Children, Dept Lab Med & Pathobiol, Lung Biol Res Programme, Toronto, ON M5G 1X8, Canada
[2] Univ Florida, Dept Pediat, Gainesville, FL USA
[3] Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA
[4] Univ Iowa, Program Gene Therapy, Dept Pediat, Carver Coll Med, Iowa City, IA USA
[5] Louisiana State Univ, Hlth Sci Ctr, Dept Med, New Orleans, LA USA
[6] Louisiana State Univ, Hlth Sci Ctr, Dept Genet, New Orleans, LA USA
[7] Childrens Hosp Pittsburgh, Dept Pediat, Pittsburgh, PA 15213 USA
[8] Univ Toronto, Hosp Sick Children, Lung Biol Res Programme, Toronto, ON M5G 1X8, Canada
[9] Univ Toronto, Dept Lab Med & Pathobiol, Toronto, ON M5G 1X8, Canada
基金
加拿大健康研究院; 美国国家卫生研究院;
关键词
TRANSMEMBRANE CONDUCTANCE REGULATOR; ADENO-ASSOCIATED VIRUS; AIRWAY EPITHELIAL-CELLS; ADULT STEM-CELLS; HUMORAL IMMUNE-RESPONSES; TARGETING TRANSGENE EXPRESSION; MONKEYS MACACA-MULATTA; GENE-TRANSFER VECTORS; MARROW-DERIVED CELLS; LONG-TERM EXPRESSION;
D O I
10.1038/sj.mt.6300002
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
Gene and cell-based therapies are considered to be potentially powerful new approaches for the management of cystic fibrosis (CF) lung disease. Despite tremendous efforts that have been made, especially in studies to understand the obstacles to gene delivery, major challenges to the application of these approaches remain to be solved. This article will review the advancements made and challenges remaining in the development of viral vector - mediated and cell-based approaches to treat patients with CF.
引用
收藏
页码:229 / 241
页数:13
相关论文
共 50 条
  • [31] Advances in Stem Cell-Based Therapies in the Treatment of Osteoarthritis
    Chen, Ye
    Cheng, Rui-Juan
    Wu, Yinlan
    Huang, Deying
    Li, Yanhong
    Liu, Yi
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2024, 25 (01)
  • [32] Nasal Epithelial Cell-Based Models for Individualized Study in Cystic Fibrosis
    Keegan, Duncan E.
    Brewington, John J.
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2021, 22 (09)
  • [33] Challenges Facing Airway Epithelial Cell-Based Therapy for Cystic Fibrosis
    Berical, Andrew
    Lee, Rhianna E.
    Randell, Scott H.
    Hawkins, Finn
    FRONTIERS IN PHARMACOLOGY, 2019, 10
  • [34] Development of advanced therapies based on viral vector-mediated overexpression of therapeutic molecules and knockdown of disease-related genes for Parkinson's disease
    Ulusoy, Ayse
    Kirik, Deniz
    THERAPEUTIC DELIVERY, 2011, 2 (01) : 37 - 50
  • [35] Repurposing therapies for the personalised treatment of cystic fibrosis
    Tosco, Antonella
    Villella, Valeria R.
    Castaldo, Alice
    Kroemer, Guido
    Maiuri, Luigi
    Raia, Valeria
    EXPERT OPINION ON ORPHAN DRUGS, 2018, 6 (06): : 361 - 373
  • [36] Cystic fibrosis, vector-mediated gene therapy, and relevance of toll-like receptors: A review of problems, progress, and possibilities
    Atkinson, Timothy J.
    CURRENT GENE THERAPY, 2008, 8 (03) : 201 - 207
  • [37] An evaluation of viral vector-mediated gene delivery to the murine neonatal brain
    Hughes, M. P.
    Howe, S. J.
    Buckley, S. M.
    Waddington, S. N.
    Rahim, A. A.
    HUMAN GENE THERAPY, 2014, 25 (11) : A107 - A107
  • [38] Activation of the CMV Promoter: Considerations for Viral Vector-Mediated Transgene Expression
    Howard, Doug
    Back, Susanne
    Dossat, Amanda
    Chen, Yun-Hsiang
    Wang, Yun
    Harvey, Brandon K.
    MOLECULAR THERAPY, 2019, 27 (04) : 161 - 161
  • [39] Production of transgenic farm animals by viral vector-mediated gene transfer
    Whitelaw, C. B. A.
    Lillico, S. G.
    King, T.
    REPRODUCTION IN DOMESTIC ANIMALS, 2008, 43 : 355 - 358
  • [40] Optimizing dendritic cell-based therapies for the treatment of mantle cell lymphoma
    Munger, Corey M.
    Vose, Julie M.
    Weisenburger, Dennis D.
    Kuszynski, Charles K.
    Joshi, Shantaram S.
    JOURNAL OF IMMUNOLOGY, 2006, 176 : S258 - S258