Gene therapy shows promise for treating Niemann-Pick disease type C1
Por um escritor misterioso
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.
Promising gene replacement therapy moves forward
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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
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