title: Advances in cell and gene-based therapies for cystic fibrosis lung disease.
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.
authors.affiliation: Department of Microbiology, Carver College of Medicine, The University of Iowa, Iowa City, Iowa 52242, USA.
authors.forename: Mayumi
authors.initials: M
authors.lastname: Oakland
authors.forename: Patrick L
authors.initials: PL
authors.lastname: Sinn
authors.forename: Paul B
authors.initials: PB
authors.lastname: McCray
mesh_terms: D000818:Animals; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D004195:Disease Models, Animal; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D033581:Stem Cell Transplantation; D016378:Tissue Transplantation