title: Lung gene therapy-How to capture illumination from the light already present in the tunnel.
abstract: 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 symptoms. Availability of techniques for identification of genetic mutations and forin vitromanipulation of genes makes it practical and attractive. After the initial hype in 1990s and later disappointments in clinical trials for more 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 strides. Clinical trials in gene therapy for retinal degenerative diseases such as Leber's congenital amaurosis (LCA) and choroideremia demonstrated clear therapeutic efficacies without apparent side effects. Although 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 medication. In addition, those success stories illuminate the path for the development of gene therapy treating other genetic diseases. Because of the differences in target organs and cells, distinct barriers to gene delivery exist in gene therapy for each genetic disease. It is not feasible for authors to review the current development in the entire field. Thus, in this article, we will focus on what we 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.
authors.affiliation: Physiology & Experimental Medicine Program, Hospital for Sick Children, University of Toronto, Toronto, Ontario M5G 0A4, Canada ; Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario M5G 0A4, Canada.
authors.forename: Emily
authors.initials: E
authors.lastname: Xia
authors.affiliation: Physiology & Experimental Medicine Program, Hospital for Sick Children, University of Toronto, Toronto, Ontario M5G 0A4, Canada ; Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario M5G 0A4, Canada.
authors.forename: Manjunatha Ankathatti
authors.initials: MA
authors.lastname: Munegowda
authors.affiliation: Physiology & Experimental Medicine Program, Hospital for Sick Children, University of Toronto, Toronto, Ontario M5G 0A4, Canada ; Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario M5G 0A4, Canada.
authors.forename: Huibi
authors.initials: H
authors.lastname: Cao
authors.affiliation: Physiology & Experimental Medicine Program, Hospital for Sick Children, University of Toronto, Toronto, Ontario M5G 0A4, Canada ; Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario M5G 0A4, Canada.
authors.forename: Jim
authors.initials: J
authors.lastname: Hu
keywords: Animal model; Cystic fibrosis; Gene therapy; Lung diseases; Vector delivery