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A tailored mouse model of CLN2 disease: A nonsense mutant for testing personalized therapies

The Neuronal Ceroid Lipofuscinoses (NCLs), also known as Batten disease, result from mutations in over a dozen genes. Although, adults are susceptible, the NCLs are frequently classified as pediatric neurodegenerative diseases due to their greater pediatric prevalence. Initial clinical presentation...

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Detalles Bibliográficos
Autores principales: Geraets, Ryan D., Langin, Logan M., Cain, Jacob T., Parker, Camille M., Beraldi, Rosanna, Kovacs, Attila D., Weimer, Jill M., Pearce, David A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5413059/
https://www.ncbi.nlm.nih.gov/pubmed/28464005
http://dx.doi.org/10.1371/journal.pone.0176526