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

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Last updated 10 novembro 2024
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.
Gene therapy shows promise for treating Niemann-Pick disease type C1
TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease
Gene therapy shows promise for treating Niemann-Pick disease type C1
Niemann–Pick Disease Type C
Gene therapy shows promise for treating Niemann-Pick disease type C1
Frontiers Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies
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
Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016
Gene therapy shows promise for treating Niemann-Pick disease type C1
Family races to raise money for research no one will fund - The Boston Globe
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
Like a tunnel for cholesterol': Scientists show how 'bad' cholesterol gets into cells
Gene therapy shows promise for treating Niemann-Pick disease type C1
Niemann-Pick disease type C as a neurovisceral disease. Schematic
Gene therapy shows promise for treating Niemann-Pick disease type C1
Enrichment of NPC1-deficient cells with the lipid LBPA stimulates autophagy, improves lysosomal function, and reduces cholesterol storage - ScienceDirect
Gene therapy shows promise for treating Niemann-Pick disease type C1
Models to study basic and applied aspects of lysosomal storage disorders - ScienceDirect
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