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注意:仅列出部分参考文献,下载原文获取全部文献信息。Therapeutic Antisense Targeting of Huntingtin
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AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease
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Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides
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Elimination of huntingtin in the adult mouse leads to progressive behavioral deficits, bilateral thalamic calcification, and altered brain iron homeostasis
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A novel neurodevelopmental disorder associated with compound heterozygous variants in the huntingtin gene
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Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease
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Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum
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Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice
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Wild-type Huntingtin protects from apoptosis upstream of caspase-3
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