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Pre Clinical Assessment of AAVrh74.MCK.GNE Viral Vector Therapeutic Potential: Robust Activity Despite Lack of Consistent Animal Model for GNE Myopathy

BACKGROUND: GNE myopathy is a unique adult onset rare neuromuscular disease caused by recessive mutations in the GNE gene. The pathophysiological mechanism of this disorder is not well understood and to date, there is no available therapy for this debilitating disease. We have previously established...

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Detalles Bibliográficos
Autores principales:	Mitrani-Rosenbaum, Stella, Yakovlev, Lena, Becker Cohen, Michal, Argov, Zohar, Fellig, Yakov, Harazi, Avi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2022
Materias:	Research Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8842764/ https://www.ncbi.nlm.nih.gov/pubmed/34806613 http://dx.doi.org/10.3233/JND-210755

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