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Hydrocephalus Complicating Intrathecal Antisense Oligonucleotide Therapy for Huntington's Disease.

作者信息

Stoker Thomas B, Andresen Katie E R, Barker Roger A

机构信息

John van Geest Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge, Cambridge, United Kingdom.

Wellcome Trust-Medical Research Council Stem Cell Institute, University of Cambridge, Cambridge, United Kingdom.

出版信息

Mov Disord. 2021 Jan;36(1):263-264. doi: 10.1002/mds.28359. Epub 2020 Oct 30.

DOI:10.1002/mds.28359
PMID:33125799
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7894279/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/49f4/7894279/af4f3f6e1666/MDS-36-263-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/49f4/7894279/af4f3f6e1666/MDS-36-263-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/49f4/7894279/af4f3f6e1666/MDS-36-263-g001.jpg

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Targeting Huntingtin Expression in Patients with Huntington's Disease.靶向亨廷顿病患者的亨廷顿蛋白表达。
N Engl J Med. 2019 Jun 13;380(24):2307-2316. doi: 10.1056/NEJMoa1900907. Epub 2019 May 6.
3
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.依库珠单抗治疗晚发性脊髓性肌萎缩症的疗效观察:一项随机、双盲、安慰剂对照 3 期试验
Alzheimers Dement. 2024 Oct;20(10):7350-7360. doi: 10.1002/alz.14166. Epub 2024 Sep 11.
4
Neuronal UBE3A substrates hold therapeutic potential for Angelman syndrome.神经UBE3A 底物为 Angelman 综合征提供了治疗潜力。
Curr Opin Neurobiol. 2024 Oct;88:102899. doi: 10.1016/j.conb.2024.102899. Epub 2024 Aug 9.
5
Preparing for Patient-Customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases.为治疗罕见病的患者定制的 N-of-1 反义寡核苷酸治疗做准备。
Genes (Basel). 2024 Jun 21;15(7):821. doi: 10.3390/genes15070821.
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Neuroimaging to Facilitate Clinical Trials in Huntington's Disease: Current Opinion from the EHDN Imaging Working Group.神经影像学在亨廷顿病临床试验中的应用:EHDN 影像学工作组的最新观点。
J Huntingtons Dis. 2024;13(2):163-199. doi: 10.3233/JHD-240016.
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