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Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy

Archive ouverte | Malerba, Alberto | CCSD

International audience

Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1

Archive ouverte | Malerba, Alberto | CCSD

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Archive ouverte | Strings-Ufombah, Vanessa | CCSD

International audience. Oculopharyngeal muscular dystrophy (OPMD) is a rare autosomal dominant disease that results from an alanine expansion in the N-terminal domain of Poly-A Binding Protein Nuclear-1 (PABPN1). We...

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