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.

Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation

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Biomolecules, Free Full-Text

Increased bacterial colonisation of Niemann–Pick disease type C1 (NPC1)

Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

Full article: Current advancements in therapy for Niemann-Pick disease: progress and pitfalls

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

Frontiers Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord

delta – Columbus Children's Foundation