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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
IJMS, Free Full-Text
Gene therapy shows promise for treating Niemann-Pick disease type C1
Marta, with Niemann Pick C, fights for a cure with gene therapy - Fundación Columbus
Gene therapy shows promise for treating Niemann-Pick disease type C1
Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
Gene therapy shows promise for treating Niemann-Pick disease type C1
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
IJMS, Free Full-Text
Gene therapy shows promise for treating Niemann-Pick disease type C1
PPT - Niemann -Pick Disease PowerPoint Presentation, free download - ID:2977714
Gene therapy shows promise for treating Niemann-Pick disease type C1
Reality of Rare Diseases: A Unique Perspective About Rare Diseases
Gene therapy shows promise for treating Niemann-Pick disease type C1
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
PDF) A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1
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