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.

Liposomal formulations for treating lysosomal storage disorders - ScienceDirect

Niemann Pick Disease News, Research

delta – Columbus Children's Foundation

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

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

Gene therapy for neurological disorders: progress and prospects

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

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

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