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Expert Opinion on Biological Therapy Volume 9, 2009 - Issue 8
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Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects

IA Pringle University of Oxford, John Radcliffe Hospital, Nuffield Department of Clinical Laboratory Sciences, Oxford, OX3 9DU, UK +44 01865 220485; +44 01865 221834; [email protected]; The UK Cystic Fibrosis Gene Therapy Consortium
,
SC Hyde University of Oxford, John Radcliffe Hospital, Nuffield Department of Clinical Laboratory Sciences, Oxford, OX3 9DU, UK +44 01865 220485; +44 01865 221834; [email protected]; The UK Cystic Fibrosis Gene Therapy Consortium
&
DR Gill University of Oxford, John Radcliffe Hospital, Nuffield Department of Clinical Laboratory Sciences, Oxford, OX3 9DU, UK +44 01865 220485; +44 01865 221834; [email protected]; The UK Cystic Fibrosis Gene Therapy Consortium
Pages 991-1003 | Published online: 23 Jun 2009

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Ranjani Somayaji, Dave P Nichols & Scott C Bell. (2020) Cystic fibrosis – Ten promising therapeutic approaches in the current era of care. Expert Opinion on Investigational Drugs 29:10, pages 1107-1124.
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Ilia Villate-Beitia, Jon Zarate, Gustavo Puras & José Luis Pedraz. (2017) Gene delivery to the lungs: pulmonary gene therapy for cystic fibrosis. Drug Development and Industrial Pharmacy 43:7, pages 1071-1081.
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Larissa Gomes dos Reis, Maree Svolos, Benedikt Hartwig, Norbert Windhab, Paul M. Young & Daniela Traini. (2017) Inhaled gene delivery: a formulation and delivery approach. Expert Opinion on Drug Delivery 14:3, pages 319-330.
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Ronak Savla & Tamara Minko. (2013) Nanotechnology approaches for inhalation treatment of fibrosis. Journal of Drug Targeting 21:10, pages 914-925.
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Stephany Y Tzeng, Peter H Yang, Warren L Grayson & Jordan J Green. (2011) Synthetic poly(ester amine) and poly(amido amine) nanoparticles for efficient DNA and siRNA delivery to human endothelial cells. International Journal of Nanomedicine 6, pages 3309-3322.
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Joana R Viola, Samir El-Andaloussi, Iulian I Oprea & CI Edvard Smith. (2010) Non-viral nanovectors for gene delivery: factors that govern successful therapeutics. Expert Opinion on Drug Delivery 7:6, pages 721-735.
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Rosy Ghanem, Véronique Laurent, Philippe Roquefort, Tanguy Haute, Sophie Ramel, Tony Le Gall, Thierry Aubry & Tristan Montier. (2020) Optimizations of In Vitro Mucus and Cell Culture Models to Better Predict In Vivo Gene Transfer in Pathological Lung Respiratory Airways: Cystic Fibrosis as an Example. Pharmaceutics 13:1, pages 47.
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Myriam Sainz-Ramos, Nuseibah AL Qtaish, Idoia Gallego, Ilia Villate- Beitia, Tania López, Gustavo Puras & José Luis Pedraz. 2019. In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders. In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders.
Laura Balcells, Cristina Fornaguera, Pau Brugada-Vilà, Marta Guerra-Rebollo, Óscar Meca-Cortés, Gema Martínez, Núria Rubio, Jerónimo Blanco, Jesús Santamaría, Anna Cascante & Salvador Borrós. (2019) SPIONs’ Enhancer Effect on Cell Transfection: An Unexpected Advantage for an Improved Gene Delivery System. ACS Omega 4:2, pages 2728-2740.
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Iwona Pranke, Anita Golec, Alexandre Hinzpeter, Aleksander Edelman & Isabelle Sermet-Gaudelus. (2019) Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine. Frontiers in Pharmacology 10.
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Gyöngyi Munkácsy, Péter Herman & Balázs Győrffy. (2019) Independent validation of induced overexpression efficiency across 242 experiments shows a success rate of 39%. Scientific Reports 9:1.
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Reto P. Bazzani, Ian A. Pringle, Mary M. Connolly, Lee A. Davies, Stephanie G. Sumner-Jones, Martin Schleef, Stephen C. Hyde & Deborah R. Gill. (2016) Transgene sequences free of CG dinucleotides lead to high level, long-term expression in the lung independent of plasmid backbone design. Biomaterials 93, pages 20-26.
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Stephany Y. Tzeng, David R. Wilson, Sarah K. Hansen, Alfredo Quiñones‐Hinojosa & Jordan J. Green. (2016) Polymeric nanoparticle‐based delivery of TRAIL DNA for cancer‐specific killing. Bioengineering & Translational Medicine 1:2, pages 149-159.
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Zhijian He, Lei Miao, Rainer Jordan, Devika S-Manickam, Robert Luxenhofer & Alexander V. Kabanov. (2015) A Low Protein Binding Cationic Poly(2-oxazoline) as Non-Viral Vector. Macromolecular Bioscience 15:7, pages 1004-1020.
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Stephanie G Sumner‐Jones & Deborah R Gill. 2005. Encyclopedia of Life Sciences. Encyclopedia of Life Sciences.
Jung Soo Suk, Anthony J. Kim, Kanika Trehan, Craig S. Schneider, Liudmila Cebotaru, Owen M. Woodward, Nicholas J. Boylan, Michael P. Boyle, Samuel K. Lai, William B. Guggino & Justin Hanes. (2014) Lung gene therapy with highly compacted DNA nanoparticles that overcome the mucus barrier. Journal of Controlled Release 178, pages 8-17.
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Wenwen Deng, Min Fu, Yue Cao, Xia Cao, Miao Wang, Yan Yang, Rui Qu, Jun Li, Ximing Xu & Jiangnan Yu. (2013) Angelica sinensis polysaccharide nanoparticles as novel non-viral carriers for gene delivery to mesenchymal stem cells. Nanomedicine: Nanotechnology, Biology and Medicine 9:8, pages 1181-1191.
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Ian A. Pringle, Stephen C. Hyde, Mary M. Connolly, Anna E. Lawton, Bihui Xu, Graciela Nunez-Alonso, Lee A. Davies, Stephanie G. Sumner-Jones & Deborah R. Gill. (2012) CpG-free plasmid expression cassettes for cystic fibrosis gene therapy. Biomaterials 33:28, pages 6833-6842.
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Diana Costa, João Queiroz, M. Graça Miguel & Björn Lindman. (2012) Swelling behavior of a new biocompatible plasmid DNA hydrogel. Colloids and Surfaces B: Biointerfaces 92, pages 106-112.
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Nicholas J. Boylan, Jung Soo Suk, Samuel K. Lai, Raz Jelinek, Michael P. Boyle, Mark J. Cooper & Justin Hanes. (2012) Highly compacted DNA nanoparticles with low MW PEG coatings: In vitro, ex vivo and in vivo evaluation. Journal of Controlled Release 157:1, pages 72-79.
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Mark A. Kay. (2011) State-of-the-art gene-based therapies: the road ahead. Nature Reviews Genetics 12:5, pages 316-328.
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P. L. Sinn, R. M. Anthony & P. B. McCray. (2011) Genetic therapies for cystic fibrosis lung disease. Human Molecular Genetics 20:R1, pages R79-R86.
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Joel C Sunshine, Corey J Bishop & Jordan J Green. (2011) Advances in polymeric and inorganic vectors for nonviral nucleic acid delivery. Therapeutic Delivery 2:4, pages 493-521.
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Uta Griesenbach & Eric W.F.W. Alton. (2011) Current Status and Future Directions of Gene and Cell Therapy for Cystic Fibrosis. BioDrugs 25:2, pages 77-88.
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Jung Soo Suk, Samuel K Lai, Nicholas J Boylan, Michelle R Dawson, Michael P Boyle & Justin Hanes. (2011) Rapid transport of muco-inert nanoparticles in cystic fibrosis sputum treated with N -acetyl cysteine . Nanomedicine 6:2, pages 365-375.
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M. Mary McMenamin. (2011) Translational benefits of gene therapy to date. Clinical Oncology and Cancer Research 8:1.
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V M Gaspar, F Sousa, J A Queiroz & I J Correia. (2011) Formulation of chitosan–TPP–pDNA nanocapsules for gene therapy applications. Nanotechnology 22:1, pages 015101.
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Asier Unciti-Broceta, Loredana Moggio, Kevin Dhaliwal, Laura Pidgeon, Keith Finlayson, Chris Haslett & Mark Bradley. (2011) Safe and efficient in vitro and in vivogene delivery: tripodal cationic lipids with programmed biodegradability. J. Mater. Chem. 21:7, pages 2154-2158.
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H. Akita, H. Hatakeyama, I.A. Khalil, Y. Yamada & H. Harashima. 2011. Comprehensive Biomaterials. Comprehensive Biomaterials 411 444 .
T. Lebhardt, S. Roesler, M. Beck-Broichsitter & T. Kissel. (2010) Polymeric nanocarriers for drug delivery to the lung. Journal of Drug Delivery Science and Technology 20:3, pages 171-180.
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Andrew M. Jones & Jennifer M. Helm. (2009) Emerging Treatments in Cystic Fibrosis. Drugs 69:14, pages 1903-1910.
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