Lung gene therapydHow to capture illumination from the light already present in the tunnel

作     者：Emily Xia Manjunatha Ankathatti Munegowda Huibi Cao Jim Hu 

作者机构：Physiology&Experimental Medicine ProgramHospital for Sick ChildrenUniversity of TorontoTorontoOntario M5G 0A4Canada Department of Laboratory Medicine and PathobiologyUniversity of TorontoTorontoOntario M5G 0A4Canada 

出 版 物：《Genes & Diseases》 (基因与疾病（英文）)

年 卷 期：2014年第1卷第1期

页      面：40-52页

学科分类：1002[医学-临床医学] 100214[医学-肿瘤学] 10[医学] 

主　　题：Cystic fibrosis Gene therapy Lung diseases Vector delivery Animal model 

摘      要：Gene therapy has been considered as the most ideal medical intervention for genetic diseases because it is intended to target the cause of diseases instead of disease *** of techniques for identification of genetic mutations and for in vitro manipulation of genes makes it practical and *** the initial hype in 1990s and later disappointments in clinical trials formore than a decade,light has finally come into the tunnel in recent years,especially in the field of eye gene therapy where it has taken big *** trials in gene therapy for retinal degenerative diseases such as Leber’s congenital amaurosis(LCA)and choroideremia demonstrated clear therapeutic efficacies without apparent side *** these successful examples are still rare and sporadic in the field,they provide the proof of concept for harnessing the power of gene therapy to treat genetic diseases and to modernize our *** addition,those success stories illuminate the path for the development of gene therapy treating other genetic *** of the differences in target organs and cells,distinct barriers to gene delivery exist in gene therapy for each genetic *** is not feasible for authors to review the current development in the entire ***,in this article,we will focus onwhatwe can learn from the current success in gene therapy for retinal degenerative diseases to speed up the gene therapy development for lung diseases,such as cystic fibrosis.