Treating Niemann-Pick C lysosomal storage: approved and emerging approaches

Journal

Trends in Molecular Medicine

ISSN

1471-4914

Date Issued

2024-12

Author(s)

KLEIN POSTERNACK, ANDRES DAVID

Facultad de Medicina Clínica Alemana Universidad del Desarrollo

Emily R. Eden

Silvana Zanlungo

Type

journal-article

DOI

10.1016/j.molmed.2024.11.011

URL

https://investigadores.udd.cl/handle/123456789/10585

Project(s)

“Identifying modifier genes and molecular pathways underlying the phenotypic variation in a lysosomal cholesterol storage disorder using a yeast systems genetics approach”

Enhanced contacts between lysosome and mitochondria as a key pathogenic pathway in neuronopathic Gaucher Disease

Subjects

lysosomal storage disorders

;

neurodegeneration

;

niemann-pick type c

;

rare disease

;

therapeutics