About the Project
We are particularly interested in applying these vectors to relevant models of Spinal muscular atrophy (SMA), a neuromuscular disorder affecting motor neurons. To study molecular mechanisms of disease and assess novel therapies we have produced iPSCs from several members of an SMA family whose symptoms are of varying severity. We are now seeking applicants to develop the iPSC-based models and test the efficacy of novel IDLVs for therapy of SMA. Project available immediately but only suitable to self-funded students or those in receipt of a personal fellowship; in addition, payment of bench fees will be required. For additional information please visit: http://pure.rhul.ac.uk/portal/en/persons/rafael-yanez_7a966e44-a812-4293-99a8-1016a6fe919a.html
Peluffo, H., Foster, E., Ahmed, S.G., Lago, N., Hutson, T., Moon, L., Yip, P., Wanisch, K., Caraballo-Miralles, V., Lladó, J., McMahon, S.B. and Yáñez-Muñoz, R.J. (2011) Efficient gene expression from non-integrating lentiviral vectors in the spinal cord. Submitted.
Wanisch, K. and Yáñez-Muñoz, R.J. (2009) Integration-deficient lentiviral vectors: a slow coming of age. Mol Ther 17, 1316-1332. doi:10.1038/mt.2009.122.
Yáñez-Muñoz, R.J., Balaggan, K.S., MacNeil, A., Howe, S., Schmidt, M., Smith, A.J., Buch, P., MacLaren, R.E., Anderson, P.N., Barker, S., Duran, Y., Bartholomae, C., von Kalle, C., Heckenlively, J.R., Kinnon, C., Ali, R.R. and Thrasher, A.J. (2006) Effective gene therapy with nonintegrating lentiviral vectors. Nat Med 12, 348-353. doi:10.1038/nm1365
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