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

Alberto Malerba; Pierre Klein; Ngoc Lu-Nguyen; Ornella Cappellari; Vanessa Strings-Ufombah; Sonal Harbaran; Peter Roelvink; David Suhy; Capucine Trollet; George Dickson

doi:10.1093/hmg/ddz167

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

Alberto Malerba, Pierre Klein, Ngoc Lu-Nguyen, Ornella Cappellari, Vanessa Strings-Ufombah, Sonal Harbaran, Peter Roelvink, David Suhy, Capucine Trollet, George Dickson

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Original language	English
Article number	ddz167
Pages (from-to)	1-24
Number of pages	24
Journal	Human Molecular Genetics
Early online date	11 Jul 2019
DOIs	https://doi.org/10.1093/hmg/ddz167
Publication status	Published - 1 Oct 2019

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10.1093/hmg/ddz167

Accepted ManuscriptAccepted author manuscript, 1.44 MB

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Towards a clinical trial for Oculopharyngeal Muscular Dystrophy
Malerba, A., Lu-Nguyen, N., Harish, P. & Dickson, G.
Association Francais contre les Myopathies
1/07/14 → 30/06/19
Project: Research

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Malerba, A., Klein, P., Lu-Nguyen, N., Cappellari, O., Strings-Ufombah, V., Harbaran, S., Roelvink, P., Suhy, D., Trollet, C., & Dickson, G. (2019). Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knock-down and replacement of mutant expanded PABPN1. Human Molecular Genetics, 1-24. [ddz167]. https://doi.org/10.1093/hmg/ddz167

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author = "Alberto Malerba and Pierre Klein and Ngoc Lu-Nguyen and Ornella Cappellari and Vanessa Strings-Ufombah and Sonal Harbaran and Peter Roelvink and David Suhy and Capucine Trollet and George Dickson",

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AU - Malerba, Alberto

AU - Klein, Pierre

AU - Lu-Nguyen, Ngoc

AU - Cappellari, Ornella

AU - Strings-Ufombah, Vanessa

AU - Harbaran, Sonal

AU - Roelvink, Peter

AU - Suhy, David

AU - Trollet, Capucine

AU - Dickson, George

PY - 2019/10/1

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DO - 10.1093/hmg/ddz167

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M1 - ddz167

ER -

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

Access to Document

Projects

Towards a clinical trial for Oculopharyngeal Muscular Dystrophy

Cite this