Chronic Enzyme Replacement to the Brain of a Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Has Differential Effects on Phenotypes of Disease

Late infantile neuronal ceroid lipofuscinosis (LINCL) is a fatal inherited neurodegenerative disease caused by loss of lysosomal protease tripeptidyl peptidase 1 (TPP1). We have investigated the effects of chronic intrathecal (IT) administration using enzyme replacement therapy (ERT) to the brain of...

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Detalles Bibliográficos
Autores principales: Wiseman, Jennifer A., Meng, Yu, Nemtsova, Yuliya, Matteson, Paul G., Millonig, James H., Moore, Dirk F., Sleat, David E., Lobel, Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363315/
https://www.ncbi.nlm.nih.gov/pubmed/28345005
http://dx.doi.org/10.1016/j.omtm.2017.01.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363315/
https://www.ncbi.nlm.nih.gov/pubmed/28345005
http://dx.doi.org/10.1016/j.omtm.2017.01.004

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