Portal de Periódicos da CAPES

A Phase I/II Study of tgAAV-CF for the Treatment of Chronic Sinusitis in Patients with Cystic Fibrosis. Stanford University, Stanford, California

1998; Mary Ann Liebert, Inc.; Volume: 9; Issue: 6 Linguagem: Inglês

10.1089/hum.1998.9.6-889

1557-7422

John A. Wagner, Mary Lynn Moran, Anna H. Messner, Richard Daifuku, C. Conrad, Thomas Reynolds, William B. Guggino, Richard B. Moss, Barrie J. Carter, Jeffrey J. Wine, Terence R. Flotte, Phyllis Gardner,

Tracheal and airway disorders

Human Gene TherapyVol. 9, No. 6 News and CommentsA Phase I/II Study of tgAAV-CF for the Treatment of Chronic Sinusitis in Patients with Cystic Fibrosis. Stanford University, Stanford, CaliforniaInvestigator(s) John A. Wagner, Mary Lynn Moran, Anna H. Messner, Richard Daifuku, Carol K. Conrad, Thomas Reynolds, William B. Guggino, Richard B. Moss, Barrie J. Carter, Jeffrey J. Wine, Terence R. Flotte, and Phyllis GardnerInvestigator(s) John A. Wagner, Mary Lynn Moran, Anna H. Messner, Richard Daifuku, Carol K. Conrad, Thomas Reynolds, William B. Guggino, Richard B. Moss, Barrie J. Carter, Jeffrey J. Wine, Terence R. Flotte, and Phyllis GardnerPublished Online:20 Mar 2008https://doi.org/10.1089/hum.1998.9.6-889AboutSectionsPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookXLinked InRedditEmail FiguresReferencesRelatedDetailsCited byGene Therapy and Cardiovascular Diseases2 December 2022Non-Modulator TherapiesClinics in Chest Medicine, Vol. 43, No. 4Medical interventions for chronic rhinosinusitis in cystic fibrosis7 April 2022 | Cochrane Database of Systematic Reviews, Vol. 2022, No. 4Gene Therapy: A Possible Alternative to CFTR Modulators?21 April 2021 | Frontiers in Pharmacology, Vol. 12Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF12 January 2021 | Pediatric Pulmonology, Vol. 56, No. S1New Directions in Pulmonary Gene Therapy Amber Vu and Paul B. McCray16 September 2020 | Human Gene Therapy, Vol. 31, No. 17-18Novel Lung Tropic Adeno-Associated Virus Capsids for Therapeutic Gene Delivery Ana Carneiro, Hyuncheol Lee, Li Lin, Joost van Haasteren, and David V. Schaffer16 September 2020 | Human Gene Therapy, Vol. 31, No. 17-18Medical interventions for chronic rhinosinusitis in cystic fibrosis23 October 2019 | Cochrane Database of Systematic Reviews, Vol. 2019, No. 10Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?Annual Review of Virology, Vol. 6, No. 1Modern pathogenesis-based methods and development of new gene and cell-based methods for cystic fibrosis treatment15 September 2018 | Genes & Cells, Vol. 13, No. 3A Preclinical Study in Rhesus Macaques for Cystic Fibrosis to Assess Gene Transfer and Transduction by AAV1 and AAV5 with a Dual-Luciferase Reporter System William B. Guggino, Janet Benson, JeanClare Seagrave, Ziying Yan, John Engelhardt, Guangping Gao, Thomas J. Conlon, and Liudmila Cebotaru1 September 2017 | Human Gene Therapy Clinical Development, Vol. 28, No. 3AAV Vector-Based Gene Therapy, Progress and Current Challenges24 May 2017Adeno-Associated Virus Gene Therapy and Its Application to the Prevention and Personalised Treatment of Rare Diseases12 July 2014Complement yourself: transcomplementation rescues partially folded mutant proteins4 March 2014 | Biophysical Reviews, Vol. 6, No. 1Birth of a New Therapeutic Platform: 47 Years of Adeno-associated Virus Biology From Virus Discovery to Licensed Gene TherapyMolecular Therapy, Vol. 21, No. 11Adenoviral Gene Transfer Corrects the Ion Transport Defect in the Sinus Epithelia of a Porcine CF ModelMolecular Therapy, Vol. 21, No. 5Sinonasal Epithelial Cell Expression of Toll-Like Receptor 9 is Elevated in Cystic Fibrosis-Associated Chronic Rhinosinusitis1 January 2013 | American Journal of Rhinology & Allergy, Vol. 27, No. 1Gene transfer: methods and applicationsGene therapy using IL-27 ameliorates Sjögren's syndrome-like autoimmune exocrinopathyArthritis Research & Therapy, Vol. 14, No. 4Adeno-associated virus for cystic fibrosis gene therapyBrazilian Journal of Medical and Biological Research, Vol. 44, No. 11Delivery of Nucleic Acids and Gene DeliveryIL-10 delivery by AAV5 vector attenuates inflammation in mice with pseudomonas pneumonia1 April 2010 | Gene Therapy, Vol. 17, No. 5Immunotherapy: rAAV2 expressing interleukin-15 inhibits HeLa cell tumor growth in mice7 May 2009 | Journal of Biomedical Science, Vol. 16, No. 1Directed evolution of adeno-associated virus to an infectious respiratory virus10 March 2009 | Proceedings of the National Academy of Sciences, Vol. 106, No. 10Genetic Modification of Adeno-Associated Viral Vector Type 2 Capsid Enhances Gene Transfer Efficiency in Polarized Human Airway Epithelial Cells April F. White, Marina Mazur, Eric J. Sorscher, Kurt R. Zinn, and Selvarangan Ponnazhagan18 December 2008 | Human Gene Therapy, Vol. 19, No. 12Apparently Nonspecific Enzyme Elevations After Portal Vein Delivery of Recombinant Adeno-Associated Virus Serotype 2 Vector in Hepatitis C Virus-Infected Chimpanzees Terence R. Flotte, Jason Goetzmann, James Caridi, Joseph Paolillo, Thomas J. Conlon, Mark Potter, Christian Mueller, and Barry J. Byrne16 June 2008 | Human Gene Therapy, Vol. 19, No. 7The Role of the Adeno-Associated Virus Capsid in Gene TransferGene delivery GAD500 autoantigen by AAV serotype 1 prevented diabetes in NOD mice: Transduction efficiency do not play important rolesImmunology Letters, Vol. 115, No. 2Activation of CFTR-specific T Cells in Cystic Fibrosis Mice Following Gene TransferMolecular Therapy, Vol. 15, No. 9Viral Vector–mediated and Cell-based Therapies for Treatment of Cystic FibrosisMolecular Therapy, Vol. 15, No. 2Unique Biologic Properties of Recombinant AAV1 Transduction in Polarized Human Airway EpitheliaJournal of Biological Chemistry, Vol. 281, No. 40Treatment of human disease by adeno-associated viral gene transfer13 April 2006 | Human Genetics, Vol. 119, No. 6Preimplantation Genetic Diagnosis28 February 2013Gene transfer: methods and applicationsAdeno-Associated Virus-Based Gene Therapy for Inherited DisordersPediatric Research, Vol. 58, No. 6Adeno-Associated Virus-Mediated Gene Transfer for Lung Diseases Terence R. Flotte16 June 2005 | Human Gene Therapy, Vol. 16, No. 6Pulmonary Transgene Expression Imaging23 April 2013Novel molecular approaches to cystic fibrosis gene therapy22 March 2005 | Biochemical Journal, Vol. 387, No. 1Progress towards gene therapy for cystic fibrosis12 May 2005 | Expert Opinion on Drug Delivery, Vol. 2, No. 2Production of research and clinical‐grade recombinant adeno‐associated virus vectorsInverted Terminal Repeat Sequences Are Important for Intermolecular Recombination and Circularization of Adeno-Associated Virus GenomesJournal of Virology, Vol. 79, No. 1Administration of a Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis16 December 2004 | Human Gene Therapy, Vol. 15, No. 11Functional Characterization of a Recombinant Adeno- Associated Virus 5-Pseudotyped Cystic Fibrosis Transmembrane Conductance Regulator Vector Jeffrey Sirninger, Christian Muller, Sofia Braag, Qiushi Tang, Hungwen Yue, Carol Detrisac, Thomas Ferkol, William B. Guggino, and Terence R. Flotte24 September 2004 | Human Gene Therapy, Vol. 15, No. 9Molecular imaging for pediatric lung diseases8 March 2004 | Pediatric Pulmonology, Vol. 37, No. 4Distinct Classes of Proteasome-Modulating Agents Cooperatively Augment Recombinant Adeno-Associated Virus Type 2 and Type 5-Mediated Transduction from the Apical Surfaces of Human Airway EpitheliaJournal of Virology, Vol. 78, No. 6Recombinant Adeno-Associated Virus As Delivery Vector for Gene Therapy—A Review Yang Lu9 July 2004 | Stem Cells and Development, Vol. 13, No. 1Lung Gene Therapy: Clinical and Regulatory Issues6 October 2004 | Clinical Research and Regulatory Affairs, Vol. 21, No. 1Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1Blood, Vol. 103, No. 1Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaquesMolecular Therapy, Vol. 8, No. 6Emerging drug treatments for cystic fibrosis2 March 2005 | Expert Opinion on Emerging Drugs, Vol. 8, No. 2Gene therapy for malignant brain tumors10 January 2014 | Expert Review of Neurotherapeutics, Vol. 3, No. 5AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia BBlood, Vol. 101, No. 8Adeno-Associated Virus —Mediated Gene Transfer for Hemophilia B1 November 2002 | International Journal of Hematology, Vol. 76, No. 4Deposition and Expression of Aerosolized rAAV Vectors in the Lungs of Rhesus MacaquesMolecular Therapy, Vol. 6, No. 4Current Status of Gene Therapy for Cystic Fibrosis Pulmonary Disease20 August 2012 | American Journal of Respiratory Medicine, Vol. 1, No. 5Production of clinical-grade recombinant adeno-associated virus vectorsCurrent Opinion in Biotechnology, Vol. 13, No. 5Use of Adeno-Associated Virus in the Treatment of Cystic Fibrosis25 June 2013Gene Therapy for _1-Antitrypsin Deficiency25 June 2013Gene Therapy: Historical Overview and Public OversightA Phase II, Double-Blind, Randomized, Placebo-Controlled Clinical Trial of tgAAVCF Using Maxillary Sinus Delivery in Patients with Cystic Fibrosis with Antrostomies John A. Wagner, Ilynn B. Nepomuceno, Anna H. Messner, Mary Lynn Moran, Eric P. Batson, Sue Dimiceli, Byron W. Brown, Julie K. Desch, Alexander M. Norbash, Carol K. Conrad, William B. Guggino, Terence R. Flotte, Jeffrey J. Wine, Barrie J. Carter, Thomas C. Reynolds, Richard B. Moss, and Phyllis Gardner6 July 2004 | Human Gene Therapy, Vol. 13, No. 11Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene (ASPA) to the Human Brain Christopher Janson, Scott McPhee, Larissa Bilaniuk, John Haselgrove, Mark Testaiuti, Andrew Freese, Dah-Jyuu Wang, David Shera, Peter Hurh, Joan Rupin, Elizabeth Saslow, Olga Goldfarb, Michael Goldberg, Ghassem Larijani, William Sharrar, Larisa Liouterman, Angelique Camp, Edwin Kolodny, Jude Samulski, and Paola Leone6 July 2004 | Human Gene Therapy, Vol. 13, No. 11Recombinant Adeno-Associated Virus Gene Therapy for Cystic Fibrosis and α1-Antitrypsin DeficiencyChest, Vol. 121, No. 3Gene Therapy for Inherited Lung Disorders: An Insight into Pulmonary DefencePulmonary Pharmacology & Therapeutics, Vol. 15, No. 1Novel approaches to the treatment of non-small cell lung cancerCritical Reviews in Oncology/Hematology, Vol. 41, No. 1Virus-Based Gene Delivery SystemsClinical Pharmacokinetics, Vol. 41, No. 12Chapter 7. Progress in the treatment of pulmonary disease in cystic fibrosisCystic Fibrosis Gene Mutations ΔF508 and 394delTT in Patients with Chronic Sinusitis in Finland8 July 2009 | Acta Oto-Laryngologica, Vol. 121, No. 8Viral Vectors for Gene TransferDrugs, Vol. 60, No. 2Gene Therapy for Lung Disease: Hype or Hope?Annals of Internal Medicine, Vol. 132, No. 8Adeno-Associated Virus: A Promising Tool for Gene DeliveryAdeno-associated virus vector-mediated gene transfer to somatic cells in the central nervous systemGene therapy for lung diseaseTrends in Pharmacological Sciences, Vol. 20, No. 6Adeno-associated virus-mediated gene deliveryThe Journal of Gene Medicine, Vol. 1, No. 3Safety and Biological Efficacy of an Adeno‐Associated Virus Vector–Cystic Fibrosis Transmembrane Regulator (AAV‐CFTR) in the Cystic Fibrosis Maxillary Sinus20 October 2009 | The Laryngoscope, Vol. 109, No. 2Maxillary sinusitis as a surrogate model for CF gene therapy clinical trials in patients with antrostomies23 November 1999 | The Journal of Gene Medicine, Vol. 1, No. 1Efficient and persistent gene transfer of AAV-CFTR in maxillary sinusThe Lancet, Vol. 351, No. 9117Prospects for Gene Therapy for the Fibrosed Heart: Targeting Regulators of Extracellular Matrix Turnover Volume 9Issue 6Apr 1998 InformationCopyright 1998, Mary Ann Liebert, Inc.To cite this article:Investigator(s) John A. Wagner, Mary Lynn Moran, Anna H. Messner, Richard Daifuku, Carol K. Conrad, Thomas Reynolds, William B. Guggino, Richard B. Moss, Barrie J. Carter, Jeffrey J. Wine, Terence R. Flotte, and Phyllis Gardner.A Phase I/II Study of tgAAV-CF for the Treatment of Chronic Sinusitis in Patients with Cystic Fibrosis. Stanford University, Stanford, California.Human Gene Therapy.Apr 1998.889-909.http://doi.org/10.1089/hum.1998.9.6-889Published in Volume: 9 Issue 6: March 20, 2008 TopicsCystic fibrosis of lungs PDF download