المؤلفون: Yanda MK; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Zeidan A; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Ciobanu C; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Izzi J; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Guggino WB; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Cebotaru L; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.

المصدر: Human gene therapy [Hum Gene Ther] 2023 Nov; Vol. 34 (21-22), pp. 1135-1144. Date of Electronic Publication: 2023 Oct 16.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: M.A. Liebert Country of Publication: United States NLM ID: 9008950 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1557-7422 (Electronic) Linking ISSN: 10430342 NLM ISO Abbreviation: Hum Gene Ther Subsets: MEDLINE

مواضيع طبية MeSH: Ferrets*/genetics , Cystic Fibrosis*, Animals ; Dependovirus/genetics ; Lung ; Liver ; Pancreas ; RNA, Messenger ; Genetic Vectors/genetics ; Transduction, Genetic

SCR Organism: Adeno-associated virus-1

المؤلفون: Yanda MK; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Tomar V; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Cebotaru CV; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Guggino WB; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Cebotaru L; Department of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.

المصدر: Human gene therapy [Hum Gene Ther] 2022 Feb; Vol. 33 (3-4), pp. 131-147. Date of Electronic Publication: 2022 Jan 07.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural

بيانات الدورية: Publisher: M.A. Liebert Country of Publication: United States NLM ID: 9008950 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1557-7422 (Electronic) Linking ISSN: 10430342 NLM ISO Abbreviation: Hum Gene Ther Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis*/genetics , Cystic Fibrosis Transmembrane Conductance Regulator*/genetics, Animals ; Dependovirus/metabolism ; Genetic Vectors/genetics ; Green Fluorescent Proteins/genetics ; Green Fluorescent Proteins/metabolism ; Macaca mulatta/genetics ; Macaca mulatta/metabolism ; Methylprednisolone/pharmacology ; Methylprednisolone/therapeutic use ; Steroids

المؤلفون: Guggino WB; Departments of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Yanda MK; Departments of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Cebotaru CV; Departments of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Cebotaru L; Departments of Medicine and Physiology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.

المصدر: Human gene therapy [Hum Gene Ther] 2020 Sep; Vol. 31 (17-18), pp. 1010-1023.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural

بيانات الدورية: Publisher: M.A. Liebert Country of Publication: United States NLM ID: 9008950 Publication Model: Print Cited Medium: Internet ISSN: 1557-7422 (Electronic) Linking ISSN: 10430342 NLM ISO Abbreviation: Hum Gene Ther Subsets: MEDLINE

مواضيع طبية MeSH: Transduction, Genetic*, Cystic Fibrosis/*therapy , Cystic Fibrosis Transmembrane Conductance Regulator/*genetics , Dependovirus/*genetics , Genetic Vectors/*administration & dosage , Lung/*metabolism, Animals ; Cystic Fibrosis/genetics ; Cystic Fibrosis/immunology ; Dose-Response Relationship, Drug ; Gene Transfer Techniques ; Genetic Therapy ; Genetic Vectors/genetics ; Genome, Viral ; Humans ; Lung/immunology ; Lung/pathology ; Macaca mulatta

المؤلفون: Guggino WB; Department of Medicine, Johns Hopkins University, Baltimore, Maryland, USA., Cebotaru L; Department of Physiology, Johns Hopkins University, Baltimore, Maryland, USA.

المصدر: Human gene therapy [Hum Gene Ther] 2020 May; Vol. 31 (9-10), pp. 538-541.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Review

بيانات الدورية: Publisher: M.A. Liebert Country of Publication: United States NLM ID: 9008950 Publication Model: Print Cited Medium: Internet ISSN: 1557-7422 (Electronic) Linking ISSN: 10430342 NLM ISO Abbreviation: Hum Gene Ther Subsets: MEDLINE

مواضيع طبية MeSH: Genetic Therapy*, Cystic Fibrosis/*genetics , Cystic Fibrosis/*therapy , Cystic Fibrosis Transmembrane Conductance Regulator/*genetics , Dependovirus/*genetics, Animals ; Gene Transfer Techniques ; Genetic Vectors ; Humans ; Promoter Regions, Genetic ; Respiratory System/virology

المؤلفون: Bergbower EAS; Departments of Medicine and Physiology, Johns Hopkins University, Baltimore, USA., Sabirzhanova I; Departments of Medicine and Physiology, Johns Hopkins University, Baltimore, USA., Boinot C; Departments of Medicine and Physiology, Johns Hopkins University, Baltimore, USA., Guggino WB; Departments of Medicine and Physiology, Johns Hopkins University, Baltimore, USA., Cebotaru L; Departments of Medicine and Physiology, Johns Hopkins University, Baltimore, USA, lcebotaru@jhmi.edu.

المصدر: Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology [Cell Physiol Biochem] 2019; Vol. 52 (6), pp. 1267-1279.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Cell Physiol Biochem Press GmbH & Co KG Country of Publication: Germany NLM ID: 9113221 Publication Model: Print Cited Medium: Internet ISSN: 1421-9778 (Electronic) Linking ISSN: 10158987 NLM ISO Abbreviation: Cell Physiol Biochem Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis/*genetics , Cystic Fibrosis Transmembrane Conductance Regulator/*genetics , Dependovirus/*genetics , Endoplasmic Reticulum/*genetics, Cell Line ; Cystic Fibrosis/therapy ; Genetic Therapy ; Humans ; Respiratory Mucosa/cytology ; Respiratory Mucosa/metabolism ; Sequence Deletion ; Transfection

المؤلفون: Liu Q; Department of Medicine, Johns Hopkins University, Baltimore, MD, USA; Department of Physiology, Johns Hopkins University, Baltimore, MD, USA., Sabirzhanova I; Department of Medicine, Johns Hopkins University, Baltimore, MD, USA; Department of Physiology, Johns Hopkins University, Baltimore, MD, USA., Yanda MK; Department of Medicine, Johns Hopkins University, Baltimore, MD, USA; Department of Physiology, Johns Hopkins University, Baltimore, MD, USA., Bergbower EAS; Department of Medicine, Johns Hopkins University, Baltimore, MD, USA; Department of Physiology, Johns Hopkins University, Baltimore, MD, USA., Boinot C; Department of Medicine, Johns Hopkins University, Baltimore, MD, USA; Department of Physiology, Johns Hopkins University, Baltimore, MD, USA., Guggino WB; Department of Physiology, Johns Hopkins University, Baltimore, MD, USA., Cebotaru L; Department of Medicine, Johns Hopkins University, Baltimore, MD, USA. Electronic address: lcebotaru@jhmi.edu.

المصدر: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2018 Sep; Vol. 17 (5), pp. 582-594. Date of Electronic Publication: 2018 Jun 20.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 101128966 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-5010 (Electronic) Linking ISSN: 15691993 NLM ISO Abbreviation: J Cyst Fibros Subsets: MEDLINE

مواضيع طبية MeSH: Aminopyridines/*pharmacology , Autophagosomes/*physiology , Benzodioxoles/*pharmacology , Cystic Fibrosis/*drug therapy , Cystic Fibrosis/*genetics , Cystic Fibrosis Transmembrane Conductance Regulator/*drug effects , Cystic Fibrosis Transmembrane Conductance Regulator/*genetics , Leucine/*analogs & derivatives, Animals ; Autophagy ; Biological Transport ; Blotting, Western ; Cell Line ; Electric Conductivity ; Leucine/pharmacology ; Mutant Proteins/drug effects ; Mutant Proteins/genetics ; Mutation ; Small Molecule Libraries/pharmacology

المؤلفون: Sabirzhanova I, Boinot C, Guggino WB, Cebotaru L

المصدر: Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology [Cell Physiol Biochem] 2018; Vol. 51 (3), pp. 1489-1499. Date of Electronic Publication: 2018 Nov 28.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Cell Physiol Biochem Press GmbH & Co KG Country of Publication: Germany NLM ID: 9113221 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1421-9778 (Electronic) Linking ISSN: 10158987 NLM ISO Abbreviation: Cell Physiol Biochem Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis/*metabolism , Cystic Fibrosis Transmembrane Conductance Regulator/*metabolism , Endoplasmic Reticulum/*metabolism , Qa-SNARE Proteins/*metabolism, Cell Line ; Cystic Fibrosis/genetics ; Cystic Fibrosis/pathology ; Cystic Fibrosis Transmembrane Conductance Regulator/analysis ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Endoplasmic Reticulum/genetics ; Endoplasmic Reticulum/pathology ; Gene Silencing ; Humans ; Protein Transport ; Proteolysis ; Qa-SNARE Proteins/analysis ; Qa-SNARE Proteins/genetics

المؤلفون: Guggino WB; a Departments of Medicine and Physiology , Johns Hopkins University , Baltimore , MD , USA., Cebotaru L; a Departments of Medicine and Physiology , Johns Hopkins University , Baltimore , MD , USA.

المصدر: Expert opinion on biological therapy [Expert Opin Biol Ther] 2017 Oct; Vol. 17 (10), pp. 1265-1273. Date of Electronic Publication: 2017 Jul 06.

نوع المنشور: Journal Article; Review

بيانات الدورية: Publisher: Taylor & Francis Country of Publication: England NLM ID: 101125414 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1744-7682 (Electronic) Linking ISSN: 14712598 NLM ISO Abbreviation: Expert Opin Biol Ther Subsets: MEDLINE

مواضيع طبية MeSH: Genetic Therapy*, Cystic Fibrosis/*therapy , Dependovirus/*genetics, Animals ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Genetic Vectors/genetics ; Genetic Vectors/metabolism ; Humans ; Promoter Regions, Genetic ; Trans-Splicing

المؤلفون: Guggino WB; 1 Department of Physiology, Johns Hopkins University , Baltimore, Maryland., Benson J; 2 Lovelace Respiratory Research Institute , Albuquerque, New Mexico., Seagrave J; 2 Lovelace Respiratory Research Institute , Albuquerque, New Mexico., Yan Z; 3 Department of Anatomy and Cell Biology, University of Iowa , Iowa City, Iowa., Engelhardt J; 3 Department of Anatomy and Cell Biology, University of Iowa , Iowa City, Iowa., Gao G; 4 Department of Microbiology & Physiological Systems, University of Massachusetts , Worcester, Massachusetts., Conlon TJ; 5 Department of Pediatrics, University of Florida , Gainesville, Florida., Cebotaru L; 6 Department of Medicine, Johns Hopkins University , Baltimore, Maryland.

المصدر: Human gene therapy. Clinical development [Hum Gene Ther Clin Dev] 2017 Sep; Vol. 28 (3), pp. 145-156. Date of Electronic Publication: 2017 Jul 19.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Mary Ann Liebert Inc. publishers Country of Publication: United States NLM ID: 101607433 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2324-8645 (Electronic) Linking ISSN: 23248637 NLM ISO Abbreviation: Hum Gene Ther Clin Dev Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis/*therapy , Dependovirus/*genetics , Genetic Therapy/*methods , Luciferases/*genetics, Animals ; Female ; Gene Transfer Techniques/adverse effects ; Genes, Reporter ; Genetic Therapy/adverse effects ; Genetic Vectors/genetics ; Luciferases/metabolism ; Macaca mulatta ; Male

المؤلفون: Veit G; Department of Physiology, McGill University, Montréal, QC H3G 1Y6, Canada., Avramescu RG; Department of Physiology, McGill University, Montréal, QC H3G 1Y6, Canada., Chiang AN; Department of Biological Sciences, University of Pittsburgh, Pittsburgh, PA 15260., Houck SA; Marsico Lung Institute, School of Medicine, University of North Carolina, Chapel Hill, NC 27514., Cai Z; School of Physiology & Pharmacology, University of Bristol, Bristol BS8 1TD, United Kingdom., Peters KW; Department of Cell Biology, School of Medicine, University of Pittsburgh, Pittsburgh, PA 15261., Hong JS; Department of Cellular, Developmental, and Integrative Biology, University of Alabama, Birmingham, AL 35294., Pollard HB; Department of Anatomy, Physiology and Genetics and Center for Medical Proteomics, Uniformed Services University of the Health Sciences, Bethesda, MD 20814., Guggino WB; Department of Physiology, Johns Hopkins University, Baltimore, MD 21205., Balch WE; Department of Chemical Physiology, Skaggs Institute of Chemical Physiology, Scripps Research Institute, La Jolla, CA 92037., Skach WR; Department of Biochemistry and Molecular Biology, Oregon Health and Science University, Portland, OR 97239., Cutting GR; McKusick-Nathans Institute of Genetic Medicine, School of Medicine, Johns Hopkins University, Baltimore, MD 21205., Frizzell RA; Department of Cell Biology, School of Medicine, University of Pittsburgh, Pittsburgh, PA 15261., Sheppard DN; School of Physiology & Pharmacology, University of Bristol, Bristol BS8 1TD, United Kingdom., Cyr DM; Marsico Lung Institute, School of Medicine, University of North Carolina, Chapel Hill, NC 27514., Sorscher EJ; Department of Pediatrics, Emory University School of Medicine, Atlanta, GA 30322., Brodsky JL; Department of Biological Sciences, University of Pittsburgh, Pittsburgh, PA 15260., Lukacs GL; Department of Physiology, McGill University, Montréal, QC H3G 1Y6, Canada Department of Biochemistry, McGill University, Montréal, QC H3G 1Y6, Canada GRASP, McGill University, Montréal, QC H3G 1Y6, Canada gergely.lukacs@mcgill.ca.

المصدر: Molecular biology of the cell [Mol Biol Cell] 2016 Feb 01; Vol. 27 (3), pp. 424-33.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: American Society for Cell Biology Country of Publication: United States NLM ID: 9201390 Publication Model: Print Cited Medium: Internet ISSN: 1939-4586 (Electronic) Linking ISSN: 10591524 NLM ISO Abbreviation: Mol Biol Cell Subsets: MEDLINE

مواضيع طبية MeSH: Cystic Fibrosis/*genetics , Cystic Fibrosis Transmembrane Conductance Regulator/*genetics, Animals ; Chloride Channel Agonists/pharmacology ; Chloride Channel Agonists/therapeutic use ; Cystic Fibrosis/classification ; Cystic Fibrosis/drug therapy ; Cystic Fibrosis Transmembrane Conductance Regulator/agonists ; Genetic Predisposition to Disease ; Humans ; Ion Channel Gating ; Mutation, Missense