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Updated: Jun 16, 2025

Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy
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Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy

Published on: June 27, 2018

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在亨廷顿病模型中,对外因子1向的miRNA减少了致病性外因子1 HTT蛋白

Marina Sogorb-Gonzalez1,2, Christian Landles3, Nicholas S Caron4

  • 1Department of Research & Development, uniQure Biopharma BV, Amsterdam 1105 BP, The Netherlands.

Brain : a journal of neurology
|August 18, 2024
PubMed
概括

使用AAV5-miHTT的基因疗法在亨廷顿病 (HD) 的小鼠模型中有效地降低了全长突变亨廷丁 (HTT) 和有毒的HTT外子1蛋白. 这种双重准的方法表明,在治疗这种致命的神经退行性疾病时,有望带来更大的治疗益处.

关键词:
在 HTT exon 1 中.在AAV5-miHTT中使用.亨廷顿病是亨廷顿病的一种疾病.亨廷顿病的小鼠模型.异常的拼接方式基因治疗的基因疗法

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相关概念视频

MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
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