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

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Descrição
NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.
Gene therapy shows promise for treating Niemann-Pick disease type C1
Gene therapy shows promise for treating Niemann-Pick disease type C1
Gene therapy shows promise for treating Niemann-Pick disease type C1
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Gene therapy shows promise for treating Niemann-Pick disease type C1
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Gene therapy shows promise for treating Niemann-Pick disease type C1
Liposomal formulations for treating lysosomal storage disorders - ScienceDirect
Gene therapy shows promise for treating Niemann-Pick disease type C1
Models to study basic and applied aspects of lysosomal storage disorders - ScienceDirect
Gene therapy shows promise for treating Niemann-Pick disease type C1
Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann–Pick Type C Using Quantitative Proteomics
Gene therapy shows promise for treating Niemann-Pick disease type C1
Diseases, Free Full-Text
Gene therapy shows promise for treating Niemann-Pick disease type C1
Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease
Gene therapy shows promise for treating Niemann-Pick disease type C1
Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
Gene therapy shows promise for treating Niemann-Pick disease type C1
Full article: Current advancements in therapy for Niemann-Pick disease: progress and pitfalls
Gene therapy shows promise for treating Niemann-Pick disease type C1
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
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