Intravitreal enzyme replacement preserves retinal structure and function in canine CLN2 neuronal ceroid lipofuscinosis

CLN2 neuronal ceroid lipofuscinosis is a hereditary neurodegenerative disorder characterized by progressive vision loss, neurological decline, and seizures. CLN2 disease results from mutations in TPP1 that encodes the lysosomal enzyme tripeptidyl peptidase-1 (TPP1). Children with CLN2 neuronal ceroi...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Exp Eye Res
Main Authors: Whiting, Rebecca E.H., Pearce, Jacqueline W., Vansteenkiste, Daniella P., Bibi, Katherine, Lim, Stefanie, Robinson, Grace O., Castaner, Leilani J., Sinclair, John, Chandra, Sundeep, Nguyen, Annalisa, O’Neill, Charles A., Katz, Martin L.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
Assuntos:
Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7484259/
https://ncbi.nlm.nih.gov/pubmed/32622066
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.exer.2020.108130
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados