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Review

Cystic fibrosis gene therapy: successes, failures and hopes for the future

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Pages 363-371 | Published online: 09 Jan 2014

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Paul N Reynolds. (2011) Gene therapy for pulmonary hypertension: prospects and challenges. Expert Opinion on Biological Therapy 11:2, pages 133-143.
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Hartmut Grasemann & Felix Ratjen. (2010) Emerging therapies for cystic fibrosis lung disease. Expert Opinion on Emerging Drugs 15:4, pages 653-659.
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James F Collawn, Lianwu Fu & Zsuzsa Bebok. (2010) Targets for cystic fibrosis therapy: proteomic analysis and correction of mutant cystic fibrosis transmembrane conductance regulator. Expert Review of Proteomics 7:4, pages 495-506.
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Beatriz Santos-Carballal, Elena Fernández Fernández & Francisco Goycoolea. (2018) Chitosan in Non-Viral Gene Delivery: Role of Structure, Characterization Methods, and Insights in Cancer and Rare Diseases Therapies. Polymers 10:4, pages 444.
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Eckhard Wolf, Alexander Kind, Bernhard Aigner & Angelika Schnieke. 2018. Animal Biotechnology 2. Animal Biotechnology 2 169 214 .
Craig S. SchneiderQingguo Xu, Nicholas J. BoylanJane ChisholmBenjamin C. Tang, Benjamin S. Schuster, Andreas HenningLaura M. EnsignEthan LeePichet AdstamongkonkulBrian W. Simons, Sho-Yu S. WangXiaoqun GongTao YuMichael P. BoyleJung Soo SukJustin Hanes. (2017) Nanoparticles that do not adhere to mucus provide uniform and long-lasting drug delivery to airways following inhalation. Science Advances 3:4.
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Elena Fernández Fernández, Nadine Bangel-Ruland, Katja Tomczak & Wolf-Michael Weber. (2016) Optimization of CFTR-mRNA transfection in human nasal epithelial cells. Translational Medicine Communications 1:1.
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Nikolai Klymiuk, Frank Seeliger, Mohammad Bohlooly-Y, Andreas Blutke, Daniel G. Rudmann & Eckhard Wolf. (2015) Tailored Pig Models for Preclinical Efficacy and Safety Testing of Targeted Therapies. Toxicologic Pathology 44:3, pages 346-357.
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Michelle Prickett & Manu Jain. 2015. Translating Gene Therapy to the Clinic. Translating Gene Therapy to the Clinic 247 260 .
Annalucia Carbone, Stefano Castellani, Maria Favia, Anna Diana, Valentina Paracchini, Sante Di Gioia, Manuela Seia, Valeria Casavola, Carla Colombo & Massimo Conese. (2014) Correction of defective CFTR / EN aC function and tightness of cystic fibrosis airway epithelium by amniotic mesenchymal stromal (stem) cells . Journal of Cellular and Molecular Medicine 18:8, pages 1631-1643.
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L E Ostrowski, W Yin, M Patel, J Sechelski, T Rogers, K Burns, B R Grubb & J C Olsen. (2014) Restoring ciliary function to differentiated primary ciliary dyskinesia cells with a lentiviral vector. Gene Therapy 21:3, pages 253-261.
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Michelle Prickett & Manu Jain. (2013) Gene therapy in cystic fibrosis. Translational Research 161:4, pages 255-264.
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Elena Cichero, Anna Basile, Maria Caterina Turco, Mauro Mazzei & Paola Fossa. (2012) Scouting new molecular targets for CFTR therapy: the HSC70/BAG-1 complex. A computational study. Medicinal Chemistry Research 21:12, pages 4430-4436.
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P. M. Barrett, A. Alagely & E. J. Topol. (2012) Cystic fibrosis in an era of genomically guided therapy. Human Molecular Genetics 21:R1, pages R66-R71.
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Diego Delgado, Alicia Rodríguez Gascón, Ana del Pozo-Rodríguez, Enrique Echevarría, Aritz Pérez Ruiz de Garibay, Juan Manuel Rodríguez & Maria Ángeles Solinís. (2012) Dextran–protamine–solid lipid nanoparticles as a non-viral vector for gene therapy: In vitro characterization and in vivo transfection after intravenous administration to mice. International Journal of Pharmaceutics 425:1-2, pages 35-43.
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Valentina Paracchini, Annalucia Carbone, Federico Colombo, Stefano Castellani, Silvia Mazzucchelli, Sante Di Gioia, Dario Degiorgio, Manuela Seia, Laura Porretti, Carla Colombo & Massimo Conese. (2012) Amniotic Mesenchymal Stem Cells: A New Source for Hepatocyte-Like Cells and Induction of CFTR Expression by Coculture with Cystic Fibrosis Airway Epithelial Cells. Journal of Biomedicine and Biotechnology 2012, pages 1-15.
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Mandy Bryon & Colin Wallis. (2011) Parents as equal partners in the paediatric cystic fibrosis multidisciplinary team. Journal of the Royal Society of Medicine 104:1_suppl, pages 30-35.
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Massimo Conese, Fiorentina Ascenzioni, A. Christopher Boyd, Charles Coutelle, Ida De Fino, Stefaan De Smedt, Joanna Rejman, Joseph Rosenecker, Dirk Schindelhauer & Bob J. Scholte. (2011) Gene and cell therapy for cystic fibrosis: From bench to bedside. Journal of Cystic Fibrosis 10, pages S114-S128.
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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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Melissa A. AshlockEric R. Olson. (2011) Therapeutics Development for Cystic Fibrosis: A Successful Model for a Multisystem Genetic Disease. Annual Review of Medicine 62:1, pages 107-125.
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Kaustubh Anil Jinturkar & Ambikanandan Misra. 2011. Challenges in Delivery of Therapeutic Genomics and Proteomics. Challenges in Delivery of Therapeutic Genomics and Proteomics 45 82 .
Elena Copreni, Lucia Palmieri, Stefano Castellani & Massimo Conese. (2010) A VSV-G Pseudotyped Last Generation Lentiviral Vector Mediates High Level and Persistent Gene Transfer in Models of Airway Epithelium In Vitro and In Vivo. Viruses 2:8, pages 1577-1588.
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