Alginate hydrogel polymers enable efficient delivery of a vascular-targeted AAV vector into aortic tissue
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Isselbacher, 2005, Thoracic and abdominal aortic aneurysms, Circulation, 111, 816, 10.1161/01.CIR.0000154569.08857.7A
El-Hamamsy, 2009, Cellular and molecular mechanisms of thoracic aortic aneurysms, Nat. Rev. Cardiol., 6, 771, 10.1038/nrcardio.2009.191
Abel, 2013, Specific gene delivery to liver sinusoidal and artery endothelial cells, Blood, 122, 2030, 10.1182/blood-2012-11-468579
Dishart, 2003, Third-generation lentivirus vectors efficiently transduce and phenotypically modify vascular cells: implications for gene therapy, J. Mol. Cell. Cardiol., 35, 739, 10.1016/S0022-2828(03)00136-6
Annoni, 2019, Modulation of immune responses in lentiviral vector-mediated gene transfer, Cell. Immunol., 342, 103802, 10.1016/j.cellimm.2018.04.012
Moiani, 2012, Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts, J. Clin. Invest., 122, 1653, 10.1172/JCI61852
Wen, 2000, Second-generation adenoviral vectors do not prevent rapid loss of transgene expression and vector DNA from the arterial wall, Arterioscler. Thromb. Vasc. Biol., 20, 1452, 10.1161/01.ATV.20.6.1452
Russell, 2017, Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial, Lancet, 390, 849, 10.1016/S0140-6736(17)31868-8
Pasi, 2020, Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A, N. Engl. J. Med., 382, 29, 10.1056/NEJMoa1908490
Al-Zaidy, 2019, From clinical trials to clinical practice: practical considerations for gene replacement therapy in SMA type 1, Pediatr. Neurol., 100, 3, 10.1016/j.pediatrneurol.2019.06.007
Feldman, 2020, Subacute liver failure following gene replacement therapy for spinal muscular atrophy type 1, J. Pediatr., 225, 252, 10.1016/j.jpeds.2020.05.044
Lee, 2012, Alginate: properties and biomedical applications, Prog. Polym. Sci., 37, 106, 10.1016/j.progpolymsci.2011.06.003
Krebs, 2010, Calcium phosphate-DNA nanoparticle gene delivery from alginate hydrogels induces in vivo osteogenesis, J. Biomed. Mater. Res. A, 92, 1131, 10.1002/jbm.a.32441
Wegman, 2011, Osteogenic differentiation as a result of BMP-2 plasmid DNA based gene therapy in vitro and in vivo, Eur. Cell. Mater., 21, 230, 10.22203/eCM.v021a18
Steinle, 2018, Incorporation of Synthetic mRNA in Injectable Chitosan-Alginate Hybrid Hydrogels for Local and Sustained Expression of Exogenous Proteins in Cells, Int. J. Mol. Sci., 19, 1313, 10.3390/ijms19051313
Krebs, 2009, Localized and sustained delivery of silencing RNA from macroscopic biopolymer hydrogels, J. Am. Chem. Soc., 131, 9204, 10.1021/ja9037615
Madrigal, 2019, Characterizing the encapsulation and release of lentivectors and adeno-associated vectors from degradable alginate hydrogels, Biomater. Sci., 7, 645, 10.1039/C8BM01218K
Hakim, 2018, AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice, JCI Insight, 3, e124297, 10.1172/jci.insight.124297
Denby, 2005, Adeno-associated virus (AAV)-7 and -8 poorly transduce vascular endothelial cells and are sensitive to proteasomal degradation, Gene Ther., 12, 1534, 10.1038/sj.gt.3302564
White, 2004, Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors, Circulation, 109, 513, 10.1161/01.CIR.0000109697.68832.5D
Work, 2004, Development of efficient viral vectors selective for vascular smooth muscle cells, Mol. Ther., 9, 198, 10.1016/j.ymthe.2003.11.006
Körbelin, 2016, A brain microvasculature endothelial cell-specific viral vector with the potential to treat neurovascular and neurological diseases, EMBO Mol. Med., 8, 609, 10.15252/emmm.201506078
Work, 2006, Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses, Mol. Ther., 13, 683, 10.1016/j.ymthe.2005.11.013
Pearce, 2019, Site-Specific Glycation and Chemo-enzymatic Antibody Sortagging for the Retargeting of rAAV6 to Inflamed Endothelium, Mol. Ther. Methods Clin. Dev., 14, 261, 10.1016/j.omtm.2019.07.003
Varadi, 2012, Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors, Gene Ther., 19, 800, 10.1038/gt.2011.143
Remes, 2020, AAV-mediated TIMP-1 overexpression in aortic tissue reduces the severity of allograft vasculopathy in mice, J. Heart Lung Transplant., 39, 389, 10.1016/j.healun.2020.01.1338
Nabel, 1989, Recombinant gene expression in vivo within endothelial cells of the arterial wall, Science, 244, 1342, 10.1126/science.2499928
Remes, 2020, Gene transfer to the vascular system: Novel translational perspectives for vascular diseases, Biochem. Pharmacol., 182, 114265, 10.1016/j.bcp.2020.114265
Sasano, 2007, Targeted high-efficiency, homogeneous myocardial gene transfer, J. Mol. Cell. Cardiol., 42, 954, 10.1016/j.yjmcc.2007.02.004
Tahlil, 1997, The Dispatch catheter as a delivery tool for arterial gene transfer, Cardiovasc. Res., 33, 181, 10.1016/S0008-6363(96)00188-5
Sinnaeve, 2002, Overexpression of a constitutively active protein kinase G mutant reduces neointima formation and in-stent restenosis, Circulation, 105, 2911, 10.1161/01.CIR.0000018169.59205.CA
Kuwabara, 2017, Tracking adventitial fibroblast contribution to disease: a review of current methods to identify resident fibroblasts, Arterioscler. Thromb. Vasc. Biol., 37, 1598, 10.1161/ATVBAHA.117.308199
Michel, 2007, Topological determinants and consequences of adventitial responses to arterial wall injury, Arterioscler. Thromb. Vasc. Biol., 27, 1259, 10.1161/ATVBAHA.106.137851
Zhou, 2018, Host responses to biomaterials and anti-inflammatory design-a brief review, Macromol. Biosci., 18, e1800112, 10.1002/mabi.201800112
Labow, 2001, Neutrophil-mediated biodegradation of medical implant materials, J. Cell. Physiol., 186, 95, 10.1002/1097-4652(200101)186:1<95::AID-JCP1008>3.0.CO;2-0
Jones, 2007, Proteomic analysis and quantification of cytokines and chemokines from biomaterial surface-adherent macrophages and foreign body giant cells, J. Biomed. Mater. Res. A, 83, 585, 10.1002/jbm.a.31221
Brodbeck, 2005, Lymphocytes and the foreign body response: lymphocyte enhancement of macrophage adhesion and fusion, J. Biomed. Mater. Res. A, 74, 222, 10.1002/jbm.a.30313
Chen, 2017, Drug-loadable calcium alginate hydrogel system for use in oral bone tissue repair, Int. J. Mol. Sci., 18, 989, 10.3390/ijms18050989
Lieder, 2013, Endotoxins-the invisible companion in biomaterials research, Tissue Eng. Part B Rev., 19, 391, 10.1089/ten.teb.2012.0636
Colella, 2017, Emerging issues in AAV-mediated ín vivo gene therapy, Mol. Ther. Methods Clin. Dev., 8, 87, 10.1016/j.omtm.2017.11.007
Martino, 2019, Immune response mechanisms against AAV vectors in animal models, Mol. Ther. Methods Clin. Dev., 17, 198, 10.1016/j.omtm.2019.12.008
Wacker, 2017, Local vascular gene therapy with apolipoprotein A-I to promote regression of atherosclerosis, Arterioscler. Thromb. Vasc. Biol., 37, 316, 10.1161/ATVBAHA.116.308258
Lai, 2017, Recombinant adeno-associated virus vector carrying the thrombomodulin lectin-like domain for the treatment of abdominal aortic aneurysm, Atherosclerosis, 262, 62, 10.1016/j.atherosclerosis.2017.03.024
Lu, 2013, Isolation and culture of smooth muscle cells from human acute type A aortic dissection, J. Cardiothorac. Surg., 8, 83, 10.1186/1749-8090-8-83
Jungmann, 2017, Protocol for efficient generation and characterization of adeno-associated viral vectors, Hum. Gene Ther. Methods, 28, 235, 10.1089/hgtb.2017.192