Large-volume Intrathecal Enzyme Delivery Increases Survival of a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

Late infantile neuronal ceroid lipofuscinosis (LINCL) is a progressive neurodegenerative lysosomal storage disorder caused by mutations in TPP1, the gene encoding the lysosomal protease tripeptidyl-peptidase (TPP1). LINCL primarily affects children, is fatal and there is no effective treatment. Admi...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Main Authors: Xu, Su, Wang, Lingling, El-Banna, Mukarram, Sohar, Istvan, Sleat, David E, Lobel, Peter
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2011
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3188738/
https://ncbi.nlm.nih.gov/pubmed/21730969
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.130
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados