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Hierarchical scaffold design for mesenchymal stem cell-based gene therapy of hemophilia B
McGill University, Montreal, Canada.
McGill University, Montreal, Canada.
Natl Res Council Canada, Boucherville, Canada.
McGill University, Montreal, Canada.
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2011 (English)In: Biomaterials, ISSN 0142-9612, E-ISSN 1878-5905, Vol. 32, no 1, 295-305 p.Article in journal (Refereed) Published
Abstract [en]

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.

Place, publisher, year, edition, pages
Elsevier, 2011. Vol. 32, no 1, 295-305 p.
National Category
Medical and Health Sciences
URN: urn:nbn:se:liu:diva-74721DOI: 10.1016/j.biomaterials.2010.08.094PubMedID: 20864158OAI: diva2:490750
Available from: 2012-02-06 Created: 2012-02-06 Last updated: 2013-12-17

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Griffith, May
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