Sortilin inhibition treats multiple neurodegenerative lysosomal storage disorders

Lysosomal storage disorders (LSDs) are a genetically and clinically diverse group of diseases characterized by lysosomal dysfunction. Batten disease is a family of severe LSDs primarily impacting the central nervous system. Here we show that AF38469, a small molecule inhibitor of sortilin, improves...

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Detalles Bibliográficos
Autores principales: Leppert, Hannah G., Anderson, Joelle T., Timm, Kaylie J., Davoli, Cristina, Pratt, Melissa A., Booth, Clarissa D., White, Katherine A., Rechtzigel, Mitchell J., Meyerink, Brandon L., Johnson, Tyler B., Brudvig, Jon J., Weimer, Jill M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10543011/
https://www.ncbi.nlm.nih.gov/pubmed/37790379
http://dx.doi.org/10.1101/2023.09.22.559064

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