Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1 - Archive ouverte HAL Accéder directement au contenu
Article Dans Une Revue Human Molecular Genetics Année : 2019

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

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hal-02466975 , version 1 (04-02-2020)

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Alberto Malerba, Pierre Klein, Ngoc Lu-Nguyen, Ornella Cappellari, Vanessa Strings-Ufombah, et al.. Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1. Human Molecular Genetics, 2019, 28 (19), pp.3301-3308. ⟨10.1093/hmg/ddz167⟩. ⟨hal-02466975⟩
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