Therapeutic approaches to Huntington disease: from the bench to the clinic.
作者： Nicholas S. Caron , E. Ray Dorsey , Michael R. Hayden
DOI: 10.1038/NRD.2018.133
关键词: Disease 、 Clinical trial 、 Clinical evaluation 、 Huntingtin 、 Neuroscience 、 Neuroinflammation 、 Medicine
摘要: … Allele-selective silencing of the mutant HTT allele is a more direct and potentially safer … Moreover, targeting the expanded CAG tract to achieve allele-selective suppression of mHTT …
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