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.

The complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease - ScienceDirect

Niemann–Pick Disease Type C: Induced Pluripotent Stem Cell–Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy - Daozhan Yu, Manju Swaroop, Mengqiao Wang, Ulrich Baxa, Rongze Yang, Yiping Yan, Turhan

Models to study basic and applied aspects of lysosomal storage disorders - ScienceDirect

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

delta – Columbus Children's Foundation

Anti-Niemann Pick C1 Single-Stranded Oligonucleotides with Locked Nucleic Acids Potently Reduce Ebola Virus Infection In Vitro: Molecular Therapy - Nucleic Acids

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

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

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

Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases