Survival Advantage of Neonatal CNS Gene Transfer for Late Infantile Neuronal Ceroid Lipofuscinosis

Late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal autosomal recessive neurodegenerative lysosomal storage disorder of childhood, is caused by mutations in the CLN2 gene, resulting in deficiency of the protein tripeptidyl peptidase I (TPP-I). We have previously shown that direct CNS admi...

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Main Authors: Sondhi, Dolan, Peterson, Daniel A., Edelstein, Andrew M., del Fierro, Katrina, Hackett, Neil R., Crystal, Ronald G.
Formato: Artigo
Idioma:Inglês
Publicado: 2008
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC2702175/
https://ncbi.nlm.nih.gov/pubmed/18639872
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.expneurol.2008.04.022
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