Gene therapy shows promise for treating Niemann-Pick disease type C1

NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.

Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease

Like a tunnel for cholesterol': Scientists show how 'bad' cholesterol gets into cells

Enrichment of NPC1-deficient cells with the lipid LBPA stimulates autophagy, improves lysosomal function, and reduces cholesterol storage - ScienceDirect

Promising gene replacement therapy moves forward

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management

Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression

IJMS, Free Full-Text

Brain gene therapy with Trojan horse lipid nanoparticles: Trends in Molecular Medicine

Community News – NNPDF

New gene therapy shows promise in reversing chronic pain