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Development of a fibrin-mediated gene delivery system for the treatment of cystinosis via design of experiment

  • Graceffa, V. Clinical development of cell therapies to halt lysosomal storage diseases: results and lessons learned. Curr. Gene Ther. https://doi.org/10.2174/1566523221666210728141924 (2021).

    Article 
    PubMed 

    Google Scholar 

  • Ariceta, G., Giordano, V. & Santos, F. Effects of long-term cysteamine treatment in patients with cystinosis. Pediatr. Nephrol. 34, 571–578 (2019).

    PubMed 

    Google Scholar 

  • Cherqui, S. Cysteamine therapy: a treatment for cystinosis, not a cure. Kidney Int. 81, 127–129 (2012).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Biswas, S. & Sornalingam, K. The ocular status of cystinosis patients receiving a hospital pharmacy-made preparation of cysteamine eye drops: a case series. Ophthalmol. Ther. 8, 125–136 (2019).

    PubMed 

    Google Scholar 

  • Liang, H., Labbé, A., Le Mouhaër, J., Plisson, C. & Baudouin, C. A new viscous cysteamine eye drops treatment for ophthalmic cystinosis: an open-label randomized comparative phase III pivotal study. Investig. Ophthalmol. Vis. Sci. 58, 2275–2283 (2017).

    CAS 

    Google Scholar 

  • U.S. Food and Drug Administration. Cystaran – FDA prescribing information, side effects and uses. 1–5 (2012)

  • Makuloluwa, A. K. & Shams, F. Cysteamine hydrochloride eye drop solution for the treatment of corneal cystine crystal deposits in patients with cystinosis: an evidence-based review. Clin. Ophthalmol. 12, 227–236 (2018).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Graceffa, V. Physical and mechanical cues affecting biomaterial-mediated plasmid DNA delivery: insights into non-viral delivery systems. J. Genet. Eng. Biotechnol. 19, 90 (2021).

    PubMed 
    PubMed Central 

    Google Scholar 

  • Yeung, A. M., Faraj, L. A., McIntosh, O. D., Dhillon, V. K. & Dua, H. S. Fibrin glue inhibits migration of ocular surface epithelial cells. Eye 30, 1389–1394 (2016).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Queiroz de Paiva, A. R., Abreu de Azevedo Fraga, L. & Torres, V. L. L. Surgical reconstruction of ocular surface tumors using fibrin sealant tissue adhesive. Ocul. Oncol. Pathol. 2, 207–211 (2016).

    PubMed 
    PubMed Central 

    Google Scholar 

  • Han, B., Schwab, I. R., Madsen, T. K. & Isseroff, R. R. A fibrin-based bioengineered ocular surface with human corneal epithelial stem cells. Cornea 21, 505–510 (2002).

    PubMed 

    Google Scholar 

  • Weisel, J. W. & Litvinov, R. I. Mechanisms of fibrin polymerization and clinical implications. Blood 121, 1712–1719 (2013).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Sacchi, V. et al. Long-lasting fibrin matrices ensure stable and functional angiogenesis by highly tunable, sustained delivery of recombinant VEGF164. Proc. Natl. Acad. Sci. U. S. A. 111, 6952–6957 (2014).

    ADS 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Lei, P., Padmashali, R. M. & Andreadis, S. T. Cell-controlled and spatially arrayed gene delivery from fibrin hydrogels. Biomaterials 30, 3790–3799 (2009).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • des Rieux, A., Shikanov, A. & Shea, L. D. Fibrin hydrogels for non-viral vector delivery in vitro. J. Control. Release 136, 148–154 (2009).

    CAS 
    PubMed 

    Google Scholar 

  • Lei, Y., Rahim, M., Ng, Q. & Segura, T. Hyaluronic acid and fibrin hydrogels with concentrated DNA/PEI polyplexes for local gene delivery. J. Control. Release 153, 255–261 (2011).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Saul, J. M., Linnes, M. P., Ratner, B. D., Giachelli, C. M. & Pun, S. H. Delivery of non-viral gene carriers from sphere-templated fibrin scaffolds for sustained transgene expression. Biomaterials 28, 4705–4716 (2007).

    CAS 
    PubMed 

    Google Scholar 

  • Shepard, J. A., Huang, A., Shikanov, A. & Shea, L. D. Balancing cell migration with matrix degradation enhances gene delivery to cells cultured three-dimensionally within hydrogels. J. Control. Release 146, 128–135 (2010).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Willerth, S. M., Arendas, K. J., Gottlieb, D. I. & Sakiyama-Elbert, S. E. Optimization of fibrin scaffolds for differentiation of murine embryonic stem cells into neural lineage cells. Biomaterials 27, 5990–6003 (2006).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Li, Y., Meng, H., Liu, Y. & Lee, B. P. Fibrin gel as an injectable biodegradable scaffold and cell carrier for tissue engineering. Sci. World J. 2015, 685690 (2015).

    Google Scholar 

  • Fernandes-Cunha, G. M. et al. In situ-forming collagen hydrogel crosslinked via multi-functional PEG as a matrix therapy for corneal defects. Sci. Rep. 10, 16671 (2020).

    ADS 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Kocatürk, T., Gençgönül, A., Balica, F., Özbağcivan, M. & Çakmak, H. Combined eye gel containing sodium hyaluronate and xanthan gum for the treatment of the corneal epithelial defect after pterygium surgery. Clin. Ophthalmol. 9, 1463–1466 (2015).

    PubMed 
    PubMed Central 

    Google Scholar 

  • Rama, P. et al. Autologous fibrin-cultured limbal stem cells permanently restore the corneal surface of patients with total limbal stem cell deficiency. Transplantation 72, 1478–1485 (2001).

    CAS 
    PubMed 

    Google Scholar 

  • Ronfard, V., Rives, J. M., Neveux, Y., Carsin, H. & Barrandon, Y. Long-term regeneration of human epidermis on third degree burns transplanted with autologous cultured epithelium grown on a fibrin matrix. Transplantation 70, 1588–1598 (2000).

    CAS 
    PubMed 

    Google Scholar 

  • Harrison, F. et al. Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis. Mol. Ther. 21, 433–444 (2013).

    CAS 
    PubMed 

    Google Scholar 

  • Lobry, T. et al. Towards a phase I clinical trial for cystinosis. Mol. Ther. 24, S14–S15 (2016).

    Google Scholar 

  • Dixon, P. & Chauhan, A. Carbon black tinted contact lenses for reduction of photophobia in cystinosis patients. Curr. Eye Res. 44, 497–504 (2019).

    CAS 
    PubMed 

    Google Scholar 

  • Liu, Z., Kompella, U. B. & Chauhan, A. Gold nanoparticle synthesis in contact lenses for drug-less ocular cystinosis treatment. Eur. J. Pharm. Biopharm. Off. J. Arbeitsgemeinschaft fur Pharm. Verfahrenstechnik e.V 165, 271–278 (2021).

    CAS 

    Google Scholar 

  • Dixon, P. et al. In vitro drug release and in vivo safety of vitamin E and cysteamine loaded contact lenses. Int. J. Pharm. 544, 380–391 (2018).

    CAS 
    PubMed 

    Google Scholar 

  • Noori, A., Ashrafi, S. J., Vaez-Ghaemi, R., Hatamian-Zaremi, A. & Webster, T. J. A review of fibrin and fibrin composites for bone tissue engineering. Int. J. Nanomedicine 12, 4937–4961 (2017).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Mühleder, S. et al. The role of fibrinolysis inhibition in engineered vascular networks derived from endothelial cells and adipose-derived stem cells. Stem Cell Res. Ther. 9, 35 (2018).

    PubMed 
    PubMed Central 

    Google Scholar 

  • Zhang, K., Fang, H., Qin, Y., Zhang, L. & Yin, J. Functionalized scaffold for in situ efficient gene transfection of mesenchymal stem cells spheroids toward chondrogenesis. ACS Appl. Mater. Interfaces 10, 33993–34004 (2018).

    CAS 
    PubMed 

    Google Scholar 

  • Kong, H. J. et al. Non-viral gene delivery regulated by stiffness of cell adhesion substrates. Nat. Mater. 4, 460–464 (2005).

    ADS 
    CAS 
    PubMed 

    Google Scholar 

  • Truong, N. F. et al. Microporous annealed particle hydrogel stiffness, void space size, and adhesion properties impact cell proliferation, cell spreading, and gene transfer. Acta Biomater. 94, 160–172 (2019).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Modaresi, S., Pacelli, S., Whitlow, J. & Paul, A. Deciphering the role of substrate stiffness in enhancing the internalization efficiency of plasmid DNA in stem cells using lipid-based nanocarriers. Nanoscale 10, 8947–8952 (2018).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Brown, A. C. & Barker, T. H. Fibrin-based biomaterials: modulation of macroscopic properties through rational design at the molecular level. Acta Biomater. 10, 1502–1514 (2014).

    CAS 
    PubMed 

    Google Scholar 

  • Ryan, E. A., Mockros, L. F., Weisel, J. W. & Lorand, L. Structural origins of fibrin clot rheology. Biophys. J. 77, 2813–2826 (1999).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Fogelson, A. L. & Keener, J. P. Toward an understanding of fibrin branching structure. Phys. Rev. E. Stat. Nonlinera Soft Matter Phys. 81, 51922 (2010).

    ADS 
    MathSciNet 

    Google Scholar 

  • Lei, Y. et al. Incorporation of active DNA/cationic polymer polyplexes into hydrogel scaffolds. Biomaterials 31, 9106–9116 (2010).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Cao, B. et al. How to optimize materials and devices via Design of Experiments and machine learning: Demonstration using organic photovoltaics. ACS Nano 12, 7434–7444 (2018).

    CAS 
    PubMed 

    Google Scholar 

  • Levin, A., Sharma, V., Hook, L. & García-Gareta, E. The importance of factorial design in tissue engineering and biomaterials science: Optimisation of cell seeding efficiency on dermal scaffolds as a case study. J. Tissue Eng. 9, 2041731418781696–2041731418781696 (2018).

    PubMed 
    PubMed Central 

    Google Scholar 

  • Arafa, M. G. & Ayoub, B. M. DOE optimization of nano-based carrier of pregabalin as hydrogel: new therapeutic & chemometric approaches for controlled d drug delivery systems. Sci. Rep. 7, 41503 (2017).

    ADS 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Xu, L. et al. Design of experiment (DoE)-driven in vitro and in vivo uptake studies of exosomes for pancreatic cancer delivery enabled by copper-free click chemistry-based labelling. J. Extracell. Vesicles 9, 1779458 (2020).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Decaris, M. L. & Leach, J. K. Design of experiments approach to engineer cell-secreted matrices for directing osteogenic differentiation. Ann. Biomed. Eng. 39, 1174–1185 (2011).

    PubMed 

    Google Scholar 

  • Kuterbekov, M. et al. Design of experiments to assess the effect of culture parameters on the osteogenic differentiation of human adipose stromal cells. Stem Cell Res. Ther. 10, 256 (2019).

    PubMed 
    PubMed Central 

    Google Scholar 

  • Naphade, S. et al. Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes. Stem Cells 33, 301–309 (2015).

    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Yeagy, B. A. et al. Kidney preservation by bone marrow cell transplantation in hereditary nephropathy. Kidney Int. 79, 1198–1206 (2011).

    CAS 
    PubMed 

    Google Scholar 

  • Syres, K. et al. Successful treatment of the murine model of cystinosis using bone marrow cell transplantation. Blood 114, 2542–2552 (2009).

    CAS 
    PubMed 

    Google Scholar 

  • Iglesias, D. M. et al. Stem cell microvesicles transfer cystinosin to human cystinotic cells and reduce cystine accumulation in vitro. PLoS ONE 7, e42840 (2012).

    ADS 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar 

  • Rocca, C. J. et al. Treatment of inherited eye defects by systemic hematopoietic stem cell transplantation. Investig. Ophthalmol. Vis. Sci. 56, 7214–7223 (2015).

    Google Scholar 

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