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
  • [1] Viral vector-mediated gene therapies
    Hollinger, Katrin
    Chamberlain, Jeffrey S.
    CURRENT OPINION IN NEUROLOGY, 2015, 28 (05) : 522 - 527
  • [2] Treatment of Cystic Fibrosis: From Gene- to Cell-Based Therapies
    Allan, Katelin M.
    Farrow, Nigel
    Donnelley, Martin
    Jaffe, Adam
    Waters, Shafagh A.
    FRONTIERS IN PHARMACOLOGY, 2021, 12
  • [3] Cell-Based Therapies for Tissue Fibrosis
    Lim, Rebecca
    Ricardo, Sharon D.
    Sievert, William
    FRONTIERS IN PHARMACOLOGY, 2017, 8
  • [4] Viral vector-mediated RNA interference
    Couto, Linda B.
    High, Katherine A.
    CURRENT OPINION IN PHARMACOLOGY, 2010, 10 (05) : 534 - 542
  • [5] Cell-based therapies for the treatment of idiopathic pulmonary fibrosis (IPF) disease
    Ghadiri, Maliheh
    Young, Paul M.
    Traini, Daniela
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2016, 16 (03) : 375 - 387
  • [6] Cell-Based Therapeutic Approaches for Cystic Fibrosis
    Duchesneau, Pascal
    Waddell, Thomas K.
    Karoubi, Golnaz
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2020, 21 (15) : 1 - 20
  • [7] Development of Cell-Based Assay to Study AAV Vector-Mediated Activation of Innate Immune Responses
    Baer, Alan
    Bhattarai, Nirjal
    MOLECULAR THERAPY, 2023, 31 (04) : 646 - 646
  • [8] Cell-Based Therapies for the Treatment of Fractures
    Borrelli, Joseph, Jr.
    Della Rocca, Gregory J.
    Schneider, Prism
    Pape, Hans-Christoph
    Cinelli, Paolo
    Weil, Yoram
    Khoury, Amal
    Liebergall, Meir
    Watson, Tracy
    JOURNAL OF ORTHOPAEDIC TRAUMA, 2019, 33 : S39 - S43
  • [9] Cell-based therapies for the treatment of schizophrenia
    Donegan, Jennifer J.
    Lodge, Daniel J.
    BRAIN RESEARCH, 2017, 1655 : 262 - 269
  • [10] Viral vector-mediated transgenic cell therapy in regenerative medicine: safety of the process
    Liu, Yang
    Wang, Dong-An
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2015, 15 (04) : 559 - 567