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- W967062177 abstract "Human Gene Therapy Clinical DevelopmentVol. 25, No. 3 Clinical ProtocolsPhase I/II Study of Intrapleural Administration of a Serotype rh.10 Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human α1-Antitrypsin cDNA to Individuals with α1-Antitrypsin DeficiencyMaria J. Chiuchiolo, Stephen M. Kaminsky, Dolan Sondhi, Denesy Mancenido, Charleen Hollmann, and Ronald G. CrystalMaria J. ChiuchioloSearch for more papers by this author, Stephen M. KaminskySearch for more papers by this author, Dolan SondhiSearch for more papers by this author, Denesy MancenidoSearch for more papers by this author, Charleen HollmannSearch for more papers by this author, and Ronald G. CrystalSearch for more papers by this authorPublished Online:19 Sep 2014https://doi.org/10.1089/humc.2014.2513AboutSectionsView articleView Full TextPDF/EPUB ToolsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail View articleFiguresReferencesRelatedDetailsCited ByOptogenetic activation of the diaphragm20 April 2022 | Scientific Reports, Vol. 12, No. 1Novel Insights into the Therapeutic Potential of Lung-Targeted Gene Transfer in the Most Common Respiratory Diseases12 March 2022 | Cells, Vol. 11, No. 6Cell and Gene Therapies for Chronic Inflammatory Lung Diseases: Emerging Technological Trends and Advancements in Respiratory Medicine24 March 2022Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo19 July 2021 | Gene Therapy, Vol. 28, No. 9Updates in the Management of Alpha-1 Antitrypsin Deficiency Lung DiseaseUS Respiratory & Pulmonary Diseases, Vol. 6, No. 1In Vivo Electroporation-Mediated, Intrahepatic Alpha1 Antitrypsin Gene Transfer Reduces Pulmonary Emphysema in Pallid Mice21 August 2020 | Pharmaceutics, Vol. 12, No. 9New Directions in Pulmonary Gene Therapy Amber Vu and Paul B. McCray16 September 2020 | Human Gene Therapy, Vol. 31, No. 17-18Design and characterization of α1-antitrypsin variants for treatment of contact system–driven thromboinflammation31 July 2019 | Blood, Vol. 134, No. 19Recent Developments in mRNA-Based Protein Supplementation Therapy to Target Lung DiseasesMolecular Therapy, Vol. 27, No. 4Muscle-Directed Gene Therapy for Alpha-1 Antitrypsin Deficiency31 March 2019Can an in vivo imaging system be used to determine localization and biodistribution of AAV5-mediated gene expression following subretinal and intravitreal delivery in mice?Experimental Eye Research, Vol. 176Lessons learned from lung and liver in-vivo gene therapy: implications for the future10 August 2018 | Expert Opinion on Biological Therapy, Vol. 18, No. 9Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin DeficiencyMolecular Therapy, Vol. 25, No. 11Genetic Modification of the Lung Directed Toward Treatment of Human Disease Dolan Sondhi, Katie M. Stiles, Bishnu P. De, and Ronald G. Crystal1 January 2017 | Human Gene Therapy, Vol. 28, No. 1Gene Therapyα1-Antitrypsin deficiency28 July 2016 | Nature Reviews Disease Primers, Vol. 2, No. 1Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung DiseaseAnnals of the American Thoracic Society, Vol. 13, No. Supplement_4Manufacturing of recombinant adeno-associated viral vectors for clinical trialsMolecular Therapy - Methods & Clinical Development, Vol. 3α 1 -antitrypsin deficiency Volume 25Issue 3Sep 2014 InformationCopyright 2014, Mary Ann Liebert, Inc.To cite this article:Maria J. Chiuchiolo, Stephen M. Kaminsky, Dolan Sondhi, Denesy Mancenido, Charleen Hollmann, and Ronald G. Crystal.Phase I/II Study of Intrapleural Administration of a Serotype rh.10 Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human α1-Antitrypsin cDNA to Individuals with α1-Antitrypsin Deficiency.Human Gene Therapy Clinical Development.Sep 2014.112-133.http://doi.org/10.1089/humc.2014.2513Published in Volume: 25 Issue 3: September 19, 2014PDF download" @default.
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- W967062177 title "Phase I/II Study of Intrapleural Administration of a Serotype rh.10 Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human α1-Antitrypsin cDNA to Individuals with α1-Antitrypsin Deficiency" @default.
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