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Hierarchical scaffo...
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Coutu, Daniel LMcGill University, Montreal, Canada
(author)
Hierarchical scaffold design for mesenchymal stem cell-based gene therapy of hemophilia B
- Article/chapterEnglish2011
Publisher, publication year, extent ...
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Elsevier,2011
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printrdacarrier
Numbers
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LIBRIS-ID:oai:DiVA.org:liu-74721
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https://urn.kb.se/resolve?urn=urn:nbn:se:liu:diva-74721URI
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https://doi.org/10.1016/j.biomaterials.2010.08.094DOI
Supplementary language notes
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Language:English
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Summary in:English
Part of subdatabase
Classification
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Subject category:ref swepub-contenttype
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Subject category:art swepub-publicationtype
Notes
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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.
Added entries (persons, corporate bodies, meetings, titles ...)
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Cuerquis, JessicaMcGill University, Montreal, Canada
(author)
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El Ayoubi, RouwaydaNatl Res Council Canada, Boucherville, Canada
(author)
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Forner, Kathy-AnnMcGill University, Montreal, Canada
(author)
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Roy, RanjanMcGill University, Montreal, Canada
(author)
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Francois, MoiraMcGill University, Montreal, Canada
(author)
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Griffith, MayOttawa Health Research Institute, Ottawa, Canada(Swepub:liu)maygr76
(author)
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Lillicrap, DavidQueen’s University, Kingston, Canada
(author)
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Yousefi, Azizeh-MitraNatl Res Council Canada, Boucherville, Canada
(author)
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Blostein, Mark DMcGill University, Montreal, Canada
(author)
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Galipeau, JacquesMcGill University, Montreal, Canada
(author)
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McGill University, Montreal, CanadaNatl Res Council Canada, Boucherville, Canada
(creator_code:org_t)
Related titles
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In:Biomaterials: Elsevier32:1, s. 295-3050142-96121878-5905
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Coutu, Daniel L
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Cuerquis, Jessic ...
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Roy, Ranjan
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Griffith, May
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Biomaterials
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Linköping University