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.

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

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

Clinical applications of gene therapy for rare diseases: A review - Papaioannou - 2023 - International Journal of Experimental Pathology - Wiley Online Library

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

Reality of Rare Diseases: A Unique Perspective About Rare Diseases

Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia: Molecular Therapy - Methods & Clinical Development

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

New gene therapy shows promise in reversing chronic pain

High incidence of unrecognized visceral/neurological late-onset Niemann-Pick disease, type C1, predicted by analysis of massively parallel sequencing data sets

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

Community News – NNPDF

Clinical applications of gene therapy for rare diseases: A review - Papaioannou - 2023 - International Journal of Experimental Pathology - Wiley Online Library

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