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Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis
The Neuronal Ceroid Lipofuscinoses (NCLs) are a family of autosomal recessive neurodegenerative disorders that annually affect 1:100,000 live births worldwide. This family of diseases results from mutations in one of 14 different genes that share common clinical and pathological etiologies. Clinical...
Autores principales: | Geraets, Ryan D., Koh, Seung yon, Hastings, Michelle L., Kielian, Tammy, Pearce, David A., Weimer, Jill M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4833901/ https://www.ncbi.nlm.nih.gov/pubmed/27083890 http://dx.doi.org/10.1186/s13023-016-0414-2 |
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