Towards a clinical trial for Oculopharyngeal Muscular Dystrophy

Project: Research

Status	Finished
Effective start/end date	1/07/14 → 30/06/19

Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knock-down and replacement of mutant expanded PABPN1
Malerba, A., Klein, P., Lu-Nguyen, N., Cappellari, O., Strings-Ufombah, V., Harbaran, S., Roelvink, P., Suhy, D., Trollet, C. & Dickson, G., 1 Oct 2019, In: Human Molecular Genetics. 28, 19, p. 3301-3308 8 p., ddz167.
Research output: Contribution to journal › Article › peer-review

Open Access
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Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD)
Harish, P., Malerba, A., Lu-Nguyen, N., Forrest, L., Cappellari, O., Roth, F., Trollet, C., Popplewell, L. & Dickson, G., Oct 2019, In: Journal of Cachexia, Sarcopenia and Muscle. 10, 5, p. 1016-1026 11 p.
Research output: Contribution to journal › Article › peer-review

Open Access
Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy
Malerba, A., Roth, F., Harish, P., Dhiab, J., Lu-Nguyen, N., Cappellari, O., Jarmin, S., Mahoudeau, A., Ythier, V., Laine, J., Negroni, E., Abgueguen, E., Simonelig, M., Guedat, P., Mouly, V., Butler-Browne, G., Voisset, C., Dickson, G. & Trollet, C., 15 May 2019, In: Human Molecular Genetics. 28, 10, p. 1694-1708 15 p., ddz007.
Research output: Contribution to journal › Article › peer-review

Open Access
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98 Downloads (Pure)

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