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Hierarchical scaffo...
Hierarchical scaffold design for mesenchymal stem cell-based gene therapy of hemophilia B
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- Coutu, Daniel L (författare)
- McGill University, Montreal, Canada
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- Cuerquis, Jessica (författare)
- McGill University, Montreal, Canada
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- El Ayoubi, Rouwayda (författare)
- Natl Res Council Canada, Boucherville, Canada
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- Forner, Kathy-Ann (författare)
- McGill University, Montreal, Canada
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- Roy, Ranjan (författare)
- McGill University, Montreal, Canada
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- Francois, Moira (författare)
- McGill University, Montreal, Canada
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- Griffith, May (författare)
- Ottawa Health Research Institute, Ottawa, Canada
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- Lillicrap, David (författare)
- Queen’s University, Kingston, Canada
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- Yousefi, Azizeh-Mitra (författare)
- Natl Res Council Canada, Boucherville, Canada
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- Blostein, Mark D (författare)
- McGill University, Montreal, Canada
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- Galipeau, Jacques (författare)
- McGill University, Montreal, Canada
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(creator_code:org_t)
- Elsevier, 2011
- 2011
- Engelska.
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Ingår i: Biomaterials. - : Elsevier. - 0142-9612 .- 1878-5905. ; 32:1, s. 295-305
- Relaterad länk:
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
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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.
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Coutu, Daniel L
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Cuerquis, Jessic ...
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El Ayoubi, Rouwa ...
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Forner, Kathy-An ...
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Roy, Ranjan
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Francois, Moira
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visa fler...
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Griffith, May
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Lillicrap, David
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Yousefi, Azizeh- ...
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Blostein, Mark D
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Galipeau, Jacque ...
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