| DOI | Trouver le DOI : https://doi.org/10.1016/j.biomaterials.2010.08.094 |
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| Auteur | Rechercher : Coutu, D.L.; Rechercher : Cuerquis, J.; Rechercher : El Ayoubi, R.1; Rechercher : Forner, K.-A.; Rechercher : Roy, R.; Rechercher : François, M.; Rechercher : Griffith, M.; Rechercher : Lillicrap, D.; Rechercher : Yousefi, A.-M.1; Rechercher : Blostein, M.D.; Rechercher : Galipeau, J. |
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| Affiliation | - Conseil national de recherches du Canada
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| Format | Texte, Article |
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| Sujet | 3D scaffolds; Cell delivery; Clinical translation; Clinical trial; Engraftment; Ex-vivo; Hemophilia; In-vivo; Mesenchymal stem cell; Nano scale; Osteogenesis; Plasma protein; Porous hydroxyapatite; Practical use; Pre-clinical; Protein delivery; Safety concerns; Self-renewal; Viral vectors; Biomineralization; Cell culture; Flowcharting; Genetic engineering; Hydroxyapatite; Ocean habitats; Proteins; Scaffolds; Stem cells; Gene therapy; biomaterial; blood clotting factor 9; calcium phosphate ceramic; collagen gel; collagen type 1; complementary DNA; green fluorescent protein; hydroxyapatite; plasma protein; polyglactin; retrovirus vector; animal cell; biomineralization; bone development; confocal microscopy; Fourier transformation; gene therapy; hemophilia; hemophilia B; histology; immunohistochemistry; infrared spectroscopy; male; mesenchymal stem cell; mouse; partial thromboplastin time; Western blotting; Calcium Phosphates; Cell Lineage; Cell Proliferation; Ceramics; Factor IX; Gene Therapy; Hemophilia B; Mesenchymal Stem Cells; Mice; Nanoparticles; Particle Size; Porosity; Tissue Scaffolds; Mus |
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| Résumé | Gene therapy for hemophilia B and other hereditary plasma protein deficiencies showed great promise in pre-clinical and early clinical trials. However, safety concerns about in vivo delivery of viral vectors and poor post-transplant survival of ex vivo modified cells remain key hurdles for clinical translation of gene therapy. We here describe a 3D scaffold system based on porous hydroxyapatite-PLGA composites coated with biomineralized collagen 1. When combined with autologous gene-engineered factor IX (hFIX) positive mesenchymal stem cells (MSCs) and implanted in hemophilic mice, these scaffolds supported long-term engraftment and systemic protein delivery by MSCs in vivo. Optimization of the scaffolds at the macro-, micro- and nanoscales provided efficient cell delivery capacity, MSC self-renewal and osteogenesis respectively, concurrent with sustained delivery of hFIX. In conclusion, the use of gene-enhanced MSC-seeded scaffolds may be of practical use for treatment of hemophilia B and other plasma protein deficiencies. © 2010 Elsevier Ltd. |
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| Date de publication | 2011 |
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| Dans | |
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| Langue | anglais |
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| Publications évaluées par des pairs | Oui |
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| Numéro NPARC | 21271727 |
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| Exporter la notice | Exporter en format RIS |
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| Signaler une correction | Signaler une correction (s'ouvre dans un nouvel onglet) |
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| Identificateur de l’enregistrement | 1b1b4fe5-e73b-4c0c-a8f1-9285b037ec3c |
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| Enregistrement créé | 2014-03-24 |
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| Enregistrement modifié | 2020-04-21 |
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