Sökning: (WFRF:(Lillicrap D.)) > Hierarchical scaffo...
Fältnamn | Indikatorer | Metadata |
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000 | 02895naa a2200373 4500 | |
001 | oai:DiVA.org:liu-74721 | |
003 | SwePub | |
008 | 120206s2011 | |||||||||||000 ||eng| | |
024 | 7 | a https://urn.kb.se/resolve?urn=urn:nbn:se:liu:diva-747212 URI |
024 | 7 | a https://doi.org/10.1016/j.biomaterials.2010.08.0942 DOI |
040 | a (SwePub)liu | |
041 | a engb eng | |
042 | 9 SwePub | |
072 | 7 | a ref2 swepub-contenttype |
072 | 7 | a art2 swepub-publicationtype |
100 | 1 | a Coutu, Daniel Lu McGill University, Montreal, Canada4 aut |
245 | 1 0 | a Hierarchical scaffold design for mesenchymal stem cell-based gene therapy of hemophilia B |
264 | 1 | b 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. | |
700 | 1 | a Cuerquis, Jessicau McGill University, Montreal, Canada4 aut |
700 | 1 | a El Ayoubi, Rouwaydau Natl Res Council Canada, Boucherville, Canada4 aut |
700 | 1 | a Forner, Kathy-Annu McGill University, Montreal, Canada4 aut |
700 | 1 | a Roy, Ranjanu McGill University, Montreal, Canada4 aut |
700 | 1 | a Francois, Moirau McGill University, Montreal, Canada4 aut |
700 | 1 | a Griffith, Mayu Ottawa Health Research Institute, Ottawa, Canada4 aut0 (Swepub:liu)maygr76 |
700 | 1 | a Lillicrap, Davidu Queen’s University, Kingston, Canada4 aut |
700 | 1 | a Yousefi, Azizeh-Mitrau Natl Res Council Canada, Boucherville, Canada4 aut |
700 | 1 | a Blostein, Mark Du McGill University, Montreal, Canada4 aut |
700 | 1 | a Galipeau, Jacquesu McGill University, Montreal, Canada4 aut |
710 | 2 | a McGill University, Montreal, Canadab Natl Res Council Canada, Boucherville, Canada4 org |
773 | 0 | t Biomaterialsd : Elsevierg 32:1, s. 295-305q 32:1<295-305x 0142-9612x 1878-5905 |
856 | 4 8 | u https://urn.kb.se/resolve?urn=urn:nbn:se:liu:diva-74721 |
856 | 4 8 | u https://doi.org/10.1016/j.biomaterials.2010.08.094 |
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