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Sökning: (WFRF:(Lillicrap D.)) > Hierarchical scaffo...

LIBRIS Formathandbok  (Information om MARC21)
FältnamnIndikatorerMetadata
00002895naa a2200373 4500
001oai:DiVA.org:liu-74721
003SwePub
008120206s2011 | |||||||||||000 ||eng|
024a https://urn.kb.se/resolve?urn=urn:nbn:se:liu:diva-747212 URI
024a https://doi.org/10.1016/j.biomaterials.2010.08.0942 DOI
040 a (SwePub)liu
041 a engb eng
042 9 SwePub
072 7a ref2 swepub-contenttype
072 7a art2 swepub-publicationtype
100a Coutu, Daniel Lu McGill University, Montreal, Canada4 aut
2451 0a Hierarchical scaffold design for mesenchymal stem cell-based gene therapy of hemophilia B
264 1b Elsevier,c 2011
338 a print2 rdacarrier
520 a 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.
700a Cuerquis, Jessicau McGill University, Montreal, Canada4 aut
700a El Ayoubi, Rouwaydau Natl Res Council Canada, Boucherville, Canada4 aut
700a Forner, Kathy-Annu McGill University, Montreal, Canada4 aut
700a Roy, Ranjanu McGill University, Montreal, Canada4 aut
700a Francois, Moirau McGill University, Montreal, Canada4 aut
700a Griffith, Mayu Ottawa Health Research Institute, Ottawa, Canada4 aut0 (Swepub:liu)maygr76
700a Lillicrap, Davidu Queen’s University, Kingston, Canada4 aut
700a Yousefi, Azizeh-Mitrau Natl Res Council Canada, Boucherville, Canada4 aut
700a Blostein, Mark Du McGill University, Montreal, Canada4 aut
700a Galipeau, Jacquesu McGill University, Montreal, Canada4 aut
710a McGill University, Montreal, Canadab Natl Res Council Canada, Boucherville, Canada4 org
773t Biomaterialsd : Elsevierg 32:1, s. 295-305q 32:1<295-305x 0142-9612x 1878-5905
8564 8u https://urn.kb.se/resolve?urn=urn:nbn:se:liu:diva-74721
8564 8u https://doi.org/10.1016/j.biomaterials.2010.08.094

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