common gene therapy viral vectors do not efficiently penetrate sputum from cystic fibrosis patients常见的基因治疗的病毒载体不有效地穿透囊性纤维化患者痰液.pdfVIP

Common Gene Therapy Viral Vectors Do Not Efficiently Penetrate Sputum from Cystic Fibrosis Patients 2 3,4 2 3 4,6 1,2,3,4,5 Kaoru Hida , Samuel K. Lai , Jung Soo Suk , Sang Y. Won , Michael P. Boyle , Justin Hanes * 1 Department of Ophthalmology, The Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America, 2 Department of Biomedical Engineering, The Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America, 3 Department of Chemical and Biomolecular Engineering, The Johns Hopkins University, Baltimore, Maryland, United States of America, 4 Institute for NanoBioTechnology, The Johns Hopkins University, Baltimore, Maryland, United States of America, 5 The Center for Nanomedicine, The Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America, 6 Division of Pulmonary and Critical Care Medicine, Johns Hopkins Adult Cystic Fibrosis Program, The Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America Abstract Norwalk virus and human papilloma virus, two viruses that infect humans at mucosal surfaces, have been found capable of rapidly penetrating human mucus secretions. Viral vectors for gene therapy of Cystic Fibrosis (CF) must similarly penetrate purulent lung airway mucus (sputum) to deliver DNA to airway epithelial cells. However, surprisingly little is known about the rates at which gene delivery vehicles penetrate sputum, including viral vectors used in clinical trials for CF gene therapy. We find that sputum spontaneously expectorated by CF patients efficiently traps two viral vectors commonly used in CF gene therapy trials, adeno