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.

delta – Columbus Children's Foundation

Heat shock protein amplification improves cerebellar myelination in the Npc1nih mouse model - eBioMedicine

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

IJMS, Free Full-Text

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

Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann–Pick Type C Using Quantitative Proteomics

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

Neurological manifestations of lysosomal disorders and emerging therapies targeting the CNS - The Lancet Child & Adolescent Health

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mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts