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.

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

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

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Advancements in Niemann-Pick Disease Type C Treatments

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

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016

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

Niemann Pick Disease News, Research

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

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

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

IJMS, Free Full-Text

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