Modeling Phenotypic Heterogeneity of Glycogen Storage Disease Type 1a Liver Disease in Mice by Somatic CRISPR/CRISPR‐associated protein 9–Mediated Gene Editing

BACKGROUND AND AIMS: Patients with glycogen storage disease type 1a (GSD‐1a) primarily present with life‐threatening hypoglycemia and display severe liver disease characterized by hepatomegaly. Despite strict dietary management, long‐term complications still occur, such as liver tumor development. V...

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Detalles Bibliográficos
Autores principales: Rutten, Martijn G.S., Derks, Terry G.J., Huijkman, Nicolette C.A., Bos, Trijnie, Kloosterhuis, Niels J., van de Kolk, Kees C.W.A., Wolters, Justina C., Koster, Mirjam H., Bongiovanni, Laura, Thomas, Rachel E., de Bruin, Alain, van de Sluis, Bart, Oosterveer, Maaike H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8597008/
https://www.ncbi.nlm.nih.gov/pubmed/34157136
http://dx.doi.org/10.1002/hep.32022

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