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Experimental Lung Research Volume 43, 2017 - Issue 9-10
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Original Articles

Gene therapy for Cystic Fibrosis: Improved delivery techniques and conditioning with lysophosphatidylcholine enhance lentiviral gene transfer in mouse lung airways

Patricia CmielewskiDepartment of Respiratory and Sleep Medicine, Women's and Children's Hospital Network, North Adelaide, SA, Australia;Robinson Research Institute, University of Adelaide, Adelaide, SA, Australia;Discipline of Paediatrics, Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA, AustraliaCorrespondence[email protected]
ORCID Iconhttps://orcid.org/0000-0002-2236-9410
ORCID Icon,
Nigel FarrowDepartment of Respiratory and Sleep Medicine, Women's and Children's Hospital Network, North Adelaide, SA, Australia;Robinson Research Institute, University of Adelaide, Adelaide, SA, Australia;Discipline of Paediatrics, Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA, Australia
,
Sharnna DevereuxDiscipline of Paediatrics, Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA, Australia
,
David ParsonsDepartment of Respiratory and Sleep Medicine, Women's and Children's Hospital Network, North Adelaide, SA, Australia;Robinson Research Institute, University of Adelaide, Adelaide, SA, Australia;Discipline of Paediatrics, Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA, Australia
&
Martin DonnelleyDepartment of Respiratory and Sleep Medicine, Women's and Children's Hospital Network, North Adelaide, SA, Australia;Robinson Research Institute, University of Adelaide, Adelaide, SA, Australia;Discipline of Paediatrics, Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA, Australia
Pages 426-433 | Received 27 Jul 2017, Accepted 19 Oct 2017, Published online: 13 Dec 2017

Citations (30)

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Read on this site (3)

Nathan Rout-Pitt, Martin Donnelley & David Parsons. (2021) In vitro optimization of miniature bronchoscope lentiviral vector delivery for the small animal lung. Experimental Lung Research 47:9, pages 417-425.
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Carina Graham & Stephen Hart. (2021) CRISPR/Cas9 gene editing therapies for cystic fibrosis. Expert Opinion on Biological Therapy 21:6, pages 767-780.
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Kamran M. Miah, Stephen C. Hyde & Deborah R. Gill. (2019) Emerging gene therapies for cystic fibrosis. Expert Review of Respiratory Medicine 13:8, pages 709-725.
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Articles from other publishers (27)

Alexandra McCarron, Patricia Cmielewski, Victoria Drysdale, David Parsons & Martin Donnelley. (2022) Effective viral-mediated lung gene therapy: is airway surface preparation necessary?. Gene Therapy 30:6, pages 469-477.
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Martin Donnelley, Patricia Cmielewski, Emma Knight, Chantelle Carpentieri, Alexandra McCarron, Nathan Rout-Pitt, David Parsons & Nigel Farrow. (2023) Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing. Gene Therapy.
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Michał Dębczyński, Damian Mojsak, Łukasz Minarowski, Monika Maciejewska, Paweł Lisowski & Robert M. Mróz. (2023) Genome-engineering technologies for modeling and treatment of cystic fibrosis. Advances in Medical Sciences 68:1, pages 111-120.
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Svetlana A. Smirnikhina. (2022) Perspectives on Genetic Medicine for Cystic Fibrosis. Current Gene Therapy 22:5, pages 386-396.
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Victoria DrysdalePatricia CmielewskiMartin Donnelley, Nicole ReyneDavid Parsons & Alexandra McCarron. (2022) Comparison of Physical Perturbation Devices for Enhancing Lentiviral Vector-Mediated Gene Transfer to the Airway Epithelium. Human Gene Therapy 33:19-20, pages 1062-1072.
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Gerry McLachlan, Eric W.F.W. Alton, A. Christopher Boyd, Nora K. Clarke, Jane C. Davies, Deborah R. GillUta Griesenbach, Jack W. Hickmott, Stephen C. HydeKamran M. MiahClaudia Juarez Molina. (2022) Progress in Respiratory Gene Therapy. Human Gene Therapy 33:17-18, pages 893-912.
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Martin Donnelley, Patricia Cmielewski, Kaye Morgan, Juliette Delhove, Nicole Reyne, Alexandra McCarron, Nathan Rout-Pitt, Victoria Drysdale, Chantelle Carpentieri, Kathryn Spiers, Akihisa Takeuchi, Kentaro Uesugi, Naoto Yagi & David Parsons. (2022) Improved in-vivo airway gene transfer via magnetic-guidance, with protocol development informed by synchrotron imaging. Scientific Reports 12:1.
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Kiran Kumar Chereddy & Lakshmi Pallavi Ganipineni. 2022. Advanced Drug Delivery Strategies for Targeting Chronic Inflammatory Lung Diseases. Advanced Drug Delivery Strategies for Targeting Chronic Inflammatory Lung Diseases 539 559 .
Chantelle Carpentieri, Nigel Farrow, Patricia Cmielewski, Nathan Rout-Pitt, Alexandra McCarron, Emma KnightDavid Parsons & Martin Donnelley. (2021) The Effects of Conditioning and Lentiviral Vector Pseudotype on Short- and Long-Term Airway Reporter Gene Expression in Mice. Human Gene Therapy 32:15-16, pages 817-827.
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Nigel Farrow, Patricia Cmielewski, Juliette Delhove, Nathan Rout-Pitt, Lewis Vaughan, Tim Kuchel, Chris Christou, John FinnieMatthew Smith, Emma Knight, Martin Donnelley & David Parsons. (2021) Towards Human Translation of Lentiviral Airway Gene Delivery for Cystic Fibrosis: A One-Month CFTR and Reporter Gene Study in Marmosets . Human Gene Therapy 32:15-16, pages 806-816.
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P. Cmielewski, J. Delhove, M. Donnelley & D. Parsons. (2021) Assessment of Lentiviral Vector Mediated CFTR Correction in Mice Using an Improved Rapid in vivo Nasal Potential Difference Measurement Protocol. Frontiers in Pharmacology 12.
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Laura I. Marquez Loza, Ashley L. Cooney, Qian Dong, Christoph O. Randak, Stefano Rivella, Patrick L. Sinn & Paul B. McCrayJr.Jr.. (2021) Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy. Molecular Therapy - Methods & Clinical Development 21, pages 94-106.
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Alexandra McCarron, Nigel Farrow, Patricia Cmielewski, Emma Knight, Martin Donnelley & David Parsons. (2021) Breaching the Delivery Barrier: Chemical and Physical Airway Epithelium Disruption Strategies for Enhancing Lentiviral-Mediated Gene Therapy. Frontiers in Pharmacology 12.
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Kübra Kaygisiz & Christopher V. Synatschke. (2020) Materials promoting viral gene delivery. Biomaterials Science 8:22, pages 6113-6156.
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Amber VuPaul B. McCray, Jr., Jr.. (2020) New Directions in Pulmonary Gene Therapy. Human Gene Therapy 31:17-18, pages 921-939.
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Siobhan Branfield & A. Valance Washington. (2020) Control the platelets, control the disease: A novel cystic fibrosis hypothesis. Journal of Thrombosis and Haemostasis 18:7, pages 1531-1534.
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Kaye Susannah Morgan, David Parsons, Patricia Cmielewski, Alexandra McCarron, Regine Gradl, Nigel Farrow, Karen Siu, Akihisa Takeuchi, Yoshio Suzuki, Kentaro Uesugi, Masayuki Uesugi, Naoto Yagi, Chris Hall, Mitzi Klein, Anton Maksimenko, Andrew Stevenson, Daniel Hausermann, Martin Dierolf, Franz Pfeiffer & Martin Donnelley. (2020) Methods for dynamic synchrotron X-ray respiratory imaging in live animals. Journal of Synchrotron Radiation 27:1, pages 164-175.
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Farzana Kastury, Euan Smith, Enzo Lombi, Martin W. Donnelley, Patricia L. Cmielewski, David W. Parsons, Matt Noerpel, Kirk G. Scheckel, Andrew M. Kingston, Glenn R. Myers, David Paterson, Martin D. de Jonge & Albert L. Juhasz. (2019) Dynamics of Lead Bioavailability and Speciation in Indoor Dust and X-ray Spectroscopic Investigation of the Link between Ingestion and Inhalation Pathways. Environmental Science & Technology 53:19, pages 11486-11495.
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Farzana Kastury, Euan Smith, Emmanuel Doelsch, Enzo Lombi, Martin Donnelley, Patricia L. Cmielewski, David W. Parsons, Kirk G. Scheckel, David Paterson, Martin D. de Jonge, Carina Herde & Albert L. Juhasz. (2019) In Vitro , in Vivo, and Spectroscopic Assessment of Lead Exposure Reduction via Ingestion and Inhalation Pathways Using Phosphate and Iron Amendments . Environmental Science & Technology 53:17, pages 10329-10341.
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Jiawen Li, Robert A. McLaughlin, Bryden C. Quirk, Juliette Delhove, Rodney W. Kirk, Alexandra McCarron, Patricia Cmielewski, Caroline Boudoux, David Parsons & Martin Donnelley. (2019) Imaging Genetically-Modified Cells with a Miniaturised Multimodal Optical Coherence Tomography + Fluorescence Probe. Imaging Genetically-Modified Cells with a Miniaturised Multimodal Optical Coherence Tomography + Fluorescence Probe.
Regine Gradl, Martin Dierolf, Lin Yang, Lorenz Hehn, Benedikt Günther, Winfried Möller, David Kutschke, Tobias Stoeger, Bernhard Gleich, Klaus Achterhold, Martin Donnelley, Franz Pfeiffer, Otmar Schmid & Kaye Susannah Morgan. (2019) Visualizing treatment delivery and deposition in mouse lungs using in vivo x-ray imaging. Journal of Controlled Release 307, pages 282-291.
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Alexandra McCarronMartin DonnelleyChantelle McIntyreDavid Parsons. (2019) Transient Lentiviral Vector Production Using a Packed-Bed Bioreactor System. Human Gene Therapy Methods 30:3, pages 93-101.
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Constanca Figueiredo, Marco Carvalho Oliveira, Chen Chen-Wacker, Katharina JanssonKlaus Höffler, Yuliia Yuzefovych, Olena Pogozhykh, Zhu Jin, Mark Kühnel, Danny Jonigk, Bettina WiegmannWiebke SommerAxel HaverichGregor WarneckeRainer Blasczyk. (2019) Immunoengineering of the Vascular Endothelium to Silence MHC Expression During Normothermic Ex Vivo Lung Perfusion . Human Gene Therapy 30:4, pages 485-496.
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Laura Marquez Loza, Eric Yuen & Paul McCrayJr.Jr.. (2019) Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease. Genes 10:3, pages 218.
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Kingsley P. Coulthard. (2018) Cystic fibrosis: novel therapies, remaining challenges. Journal of Pharmacy Practice and Research 48:6, pages 569-577.
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Martin Donnelley & David W. Parsons. (2018) Gene Therapy for Cystic Fibrosis Lung Disease: Overcoming the Barriers to Translation to the Clinic. Frontiers in Pharmacology 9.
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Chantelle McIntyreMartin DonnelleyNathan Rout-PittDavid Parsons. (2018) Lobe-Specific Gene Vector Delivery to Rat Lungs Using a Miniature Bronchoscope. Human Gene Therapy Methods 29:5, pages 228-235.
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