细胞和基因治疗在囊性纤维化肺病中的进展。
Advances in cell and gene-based therapies for cystic fibrosis lung disease.
机构信息
Department of Microbiology, Carver College of Medicine, The University of Iowa, Iowa City, Iowa 52242, USA.
出版信息
Mol Ther. 2012 Jun;20(6):1108-15. doi: 10.1038/mt.2012.32. Epub 2012 Feb 28.
Abstract
Cystic fibrosis (CF) is a disease characterized by airway infection, inflammation, remodeling, and obstruction that gradually destroy the lungs. Direct delivery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway epithelia may offer advantages, as the tissue is accessible for topical delivery of vectors. Yet, physical and host immune barriers in the lung present challenges for successful gene transfer to the respiratory tract. Advances in gene transfer approaches, tissue engineering, and novel animal models are generating excitement within the CF research field. This review discusses current challenges and advancements in viral and nonviral vectors, cell-based therapies, and CF animal models.
摘要
囊性纤维化（CF）是一种以气道感染、炎症、重塑和阻塞为特征的疾病，这些逐渐破坏肺部。将囊性纤维化跨膜电导调节因子（CFTR）基因直接递送至气道上皮可能具有优势，因为组织可用于递呈载体的局部给药。然而，肺部的物理和宿主免疫屏障给呼吸道的成功基因转移带来了挑战。基因转移方法、组织工程和新型动物模型的进步在 CF 研究领域引起了兴奋。本文讨论了病毒和非病毒载体、基于细胞的疗法以及 CF 动物模型的当前挑战和进展。
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