An Adeno-Associated Viral Vector Capable of Penetrating the Mucus Barrier to Inhaled Gene Therapy

Molecular Therapy - Methods & Clinical Development - Tập 9 - Trang 296-304 - 2018
Gregg A. Duncan1,2,3, Nam‐Ho Kim1,4, Yanerys Colon-Cortes5, Jason Rodriguez1,2, Marina Mazur6, Susan E. Birket6, Steven M. Rowe6, Natalie E. West7, Alessandra Livraghi-Butrico8, Richard C. Boucher8, Justin Hanes1,4,2,9, George Aslanidi5,10, Jung Soo Suk1,2
1Center for Nanomedicine, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA
2Department of Ophthalmology, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA
3Fischell Department of Bioengineering, University of Maryland, College Park, MD 20742, USA
4Department of Chemical and Biomolecular Engineering, Johns Hopkins University, Baltimore, MD 21218, USA
5Department of Pediatrics , University of Florida, Gainesville, FL 32610 USA
6Department of Medicine, Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL 35294, USA
7Department of Medicine, Division of Pulmonary and Critical Care Medicine, Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA
8Marsico Lung Institute/Cystic Fibrosis Research Center, The University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC 27599, USA
9Departments of Biomedical Engineering, Environmental and Health Sciences, Oncology, Neurosurgery, and Pharmacology and Molecular Sciences, Johns Hopkins University, Baltimore, MD 21287, USA
10The Hormel Institute, University of Minnesota, Austin, MN 55912, USA

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Griesenbach, 2009, Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy, Adv. Drug Deliv. Rev., 61, 128, 10.1016/j.addr.2008.09.010

Limberis, 2009, Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro, Mol. Ther., 17, 294, 10.1038/mt.2008.261

Yan, 2013, A novel chimeric adenoassociated virus 2/human bocavirus 1 parvovirus vector efficiently transduces human airway epithelia, Mol. Ther, 21, 2181, 10.1038/mt.2013.92

Cooney, 2016, Lentiviral-mediated phenotypic correction of cystic fibrosis pigs, JCI Insight, 1, e88730, 10.1172/jci.insight.88730

Steines, 2016, CFTR gene transfer with AAV improves early cystic fibrosis pig phenotypes, JCI Insight, 1, e88728, 10.1172/jci.insight.88728

Flotte, 2010, Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway, Mol. Ther, 18, 594, 10.1038/mt.2009.230

Yan, 2017, Human bocavirus type-1 capsid facilitates the transduction of ferret airways by adeno-associated virus genomes, Hum. Gene Ther., 28, 612, 10.1089/hum.2017.060

Griesenbach, 2012, Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy, Am. J. Respir. Crit. Care Med., 186, 846, 10.1164/rccm.201206-1056OC

Alton, 2017, Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis, Thorax, 72, 137, 10.1136/thoraxjnl-2016-208406

Excoffon, 2009, Directed evolution of adeno-associated virus to an infectious respiratory virus, Proc. Natl. Acad. Sci. USA, 106, 3865, 10.1073/pnas.0813365106

Gernoux, 2017, Regulatory and exhausted T cell responses to AAV capsid, Hum. Gene Ther., 28, 338, 10.1089/hum.2017.022

Tse, 2017, Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion, Proc. Natl. Acad. Sci. USA, 114, E4812, 10.1073/pnas.1704766114

Cone, 2009, Barrier properties of mucus, Adv. Drug Deliv. Rev., 61, 75, 10.1016/j.addr.2008.09.008

Duncan, 2016, The mucus barrier to inhaled gene therapy, Mol. Ther., 24, 2043, 10.1038/mt.2016.182

Ferrari, 2002, Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis, Adv. Drug Deliv. Rev., 54, 1373, 10.1016/S0169-409X(02)00145-X

Kim, 2016, Barriers to inhaled gene therapy of obstructive lung diseases: A review, J. Control. Release, 240, 465, 10.1016/j.jconrel.2016.05.031

Mastorakos, 2015, Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy, Proc. Natl. Acad. Sci. USA, 112, 8720, 10.1073/pnas.1502281112

Schneider, 2017, Nanoparticles that do not adhere to mucus provide uniform and long-lasting drug delivery to airways following inhalation, Sci. Adv., 3, e1601556, 10.1126/sciadv.1601556

Suk, 2014, Lung gene therapy with highly compacted DNA nanoparticles that overcome the mucus barrier, J. Control. Release, 178, 8, 10.1016/j.jconrel.2014.01.007

Suk, 2016, Could recent advances in DNA-loaded nanoparticles lead to effective inhaled gene therapies?, Nanomedicine (Lond.), 11, 193, 10.2217/nnm.15.194

Forier, 2013, Transport of nanoparticles in cystic fibrosis sputum and bacterial biofilms by single-particle tracking microscopy, Nanomedicine (Lond.), 8, 935, 10.2217/nnm.12.129

Fahy, 2010, Airway mucus function and dysfunction, N. Engl. J. Med., 363, 2233, 10.1056/NEJMra0910061

Knowles, 2002, Mucus clearance as a primary innate defense mechanism for mammalian airways, J. Clin. Invest., 109, 571, 10.1172/JCI0215217

Hida, 2011, Common gene therapy viral vectors do not efficiently penetrate sputum from cystic fibrosis patients, PLoS ONE, 6, e19919, 10.1371/journal.pone.0019919

Schuster, 2014, Overcoming the cystic fibrosis sputum barrier to leading adeno-associated virus gene therapy vectors, Mol. Ther., 22, 1484, 10.1038/mt.2014.89

Li, 2011, AAV-6 mediated efficient transduction of mouse lower airways, Virology, 417, 327, 10.1016/j.virol.2011.06.009

Kurosaki, 2017, Optimization of adeno-associated virus vector-mediated gene transfer to the respiratory tract, Gene Ther., 24, 290, 10.1038/gt.2017.19

McClain, 2016, Vector serotype screening for use in ovine perinatal lung gene therapy, J. Pediatr. Surg., 51, 879, 10.1016/j.jpedsurg.2016.02.048

Liu, 2007, Biological differences in rAAV transduction of airway epithelia in humans and in old world non-human primates, Mol. Ther., 15, 2114, 10.1038/sj.mt.6300277

Liu, 2007, Comparative biology of rAAV transduction in ferret, pig and human airway epithelia, Gene Ther., 14, 1543, 10.1038/sj.gt.3303014

Duncan, 2016, Microstructural alterations of sputum in cystic fibrosis lung disease, JCI Insight, 1, e88198, 10.1172/jci.insight.88198

Henderson, 2014, Cystic fibrosis airway secretions exhibit mucin hyperconcentration and increased osmotic pressure, J. Clin. Invest., 124, 3047, 10.1172/JCI73469

Schuster, 2015, Particle tracking in drug and gene delivery research: state-of-the-art applications and methods, Adv. Drug Deliv. Rev., 91, 70, 10.1016/j.addr.2015.03.017

Wu, 2006, Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes, J. Virol., 80, 11393, 10.1128/JVI.01288-06

Sagel, 2007, Sputum biomarkers of inflammation in cystic fibrosis lung disease, Proc. Am. Thorac. Soc., 4, 406, 10.1513/pats.200703-044BR

Ribeiro, 2009, Azithromycin treatment alters gene expression in inflammatory, lipid metabolism, and cell cycle pathways in well-differentiated human airway epithelia, PLoS ONE, 4, e5806, 10.1371/journal.pone.0005806

Mall, 2004, Increased airway epithelial Na+ absorption produces cystic fibrosis-like lung disease in mice, Nat. Med., 10, 487, 10.1038/nm1028

Mall, 2008, Development of chronic bronchitis and emphysema in beta-epithelial Na+ channel-overexpressing mice, Am. J. Respir. Crit. Care Med., 177, 730, 10.1164/rccm.200708-1233OC

Huang, 2017, Protein nanocages that penetrate airway mucus and tumor tissue, Proc. Natl. Acad. Sci. USA, 114, E6595, 10.1073/pnas.1705407114

Zhang, 2009, CFTR delivery to 25% of surface epithelial cells restores normal rates of mucus transport to human cystic fibrosis airway epithelium, PLoS Biol., 7, e1000155, 10.1371/journal.pbio.1000155

Wu, 2006, α2,3 and α2,6 N-linked sialic acids facilitate efficient binding and transduction by adeno-associated virus types 1 and 6, J. Virol., 80, 9093, 10.1128/JVI.00895-06

Sanders, 2000, Cystic fibrosis sputum: a barrier to the transport of nanospheres, Am. J. Respir. Crit. Care Med., 162, 1905, 10.1164/ajrccm.162.5.9909009

Suk, 2011, N-acetylcysteine enhances cystic fibrosis sputum penetration and airway gene transfer by highly compacted DNA nanoparticles, Mol. Ther., 19, 1981, 10.1038/mt.2011.160

Schuster, 2013, Nanoparticle diffusion in respiratory mucus from humans without lung disease, Biomaterials, 34, 3439, 10.1016/j.biomaterials.2013.01.064

Kesimer, 2017, Airway mucin concentration as a marker of chronic bronchitis, N. Engl. J. Med., 377, 911, 10.1056/NEJMoa1701632

Xie, 2011, Structure-function analysis of receptor-binding in adeno-associated virus serotype 6 (AAV-6), Virology, 420, 10, 10.1016/j.virol.2011.08.011

Bals, 1999, Transduction of well-differentiated airway epithelium by recombinant adeno-associated virus is limited by vector entry, J. Virol., 73, 6085, 10.1128/JVI.73.7.6085-6088.1999

Yan, 2004, Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia, J. Virol., 78, 2863, 10.1128/JVI.78.6.2863-2874.2004

Mitomo, 2010, Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes, Mol. Ther., 18, 1173, 10.1038/mt.2010.13

Aslanidi, 2013, Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold?, PLoS ONE, 8, e59142, 10.1371/journal.pone.0059142

Pandya, 2014, Rationally designed capsid and transgene cassette of AAV6 vectors for dendritic cell-based cancer immunotherapy, Immunol. Cell Biol., 92, 116, 10.1038/icb.2013.74

Sayroo, 2016, Development of novel AAV serotype 6 based vectors with selective tropism for human cancer cells, Gene Ther., 23, 18, 10.1038/gt.2015.89

Cozens, 1992, Characterization of immortal cystic fibrosis tracheobronchial gland epithelial cells, Proc. Natl. Acad. Sci. USA, 89, 5171, 10.1073/pnas.89.11.5171

Horani, 2013, Rho-associated protein kinase inhibition enhances airway epithelial Basal-cell proliferation and lentivirus transduction, Am. J. Respir. Cell Mol. Biol., 49, 341, 10.1165/rcmb.2013-0046TE

Suprynowicz, 2012, Conditionally reprogrammed cells represent a stem-like state of adult epithelial cells, Proc. Natl. Acad. Sci. USA, 109, 20035, 10.1073/pnas.1213241109

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Molecular Therapy - Methods & Clinical Development

Tập 9

296-304

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