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'基因靜默'技術為阿茲海默症患者提供新希望

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Researchers have come up with a novel therapy to eliminate the harmful tau protein, which is primarily responsible for Alzheimer's disease.

研究人員們已經提出一種,消除主要導致阿茲海默症之有害tau蛋白的新療法。

 

A first-of-its-kind research trial conducted collaboratively by scientists at the University College London and University College London Hospitals found a "gene silencing" technique that can reduce the risk of dementia and Alzheimer's disease, as per the Science Blog.

根據《科學部落格》的報道,由英國倫敦大學學院及倫敦大學學院醫院之科學家們合作進行的一項,史無前例之研究試驗發現了一種,能降低癡呆症及阿茲海默症風險的"基因靜默"技術。

 

To carry out the process, a drug called BIIB080 (MAPTRx) was employed to "silence" the gene coding for the tau protein, which was formed by the microtubule-associated protein tau (MAPT) gene. Through the method, the gene was deterred from being translated into the protein.

為了完成此過程,使用了一種被稱為BIIB080 (MAPTRx)的藥物來"靜默",為該種tau蛋白指定遺傳碼的基因。該蛋白由,與微管相關的蛋白tau (MAPT)基因所產生。經由此種方法,該基因被阻止轉譯成蛋白質。

 

The method will also make sure that lesser amounts of tau protein are produced so that the disease doesn't present itself in real-time, the Science Blog reported.

The findings of the first installment of the research have been published in Nature Medicine. In a cohort study involving 46 participants, the method showed positive outcomes and significantly lowered the amount of tau in the brain.

《科學部落格》報導了,該方法也能確保,產生較少量的tau蛋白。因此,該種疾病不會即時出現。該項研究的第一期結果,已經被發表於《自然•醫學》期刊。在一項涉及46名參與者的群組研究中,該種方法展現了正面結果,且顯著降低大腦中的tau數量。

 

The protein is found in the nerve cells of the brain. At the onset of Alzheimer's, the protein increases by abnormal measures and creates neurofibrillary tangles inside the brain's neurons. Compared to a healthy brain, those with Alzheimer's see their functioning disrupted by the tangles, which ultimately results in cognitive impairment.

該種蛋白,於大腦的神經細胞中被發現。於阿茲海默症開始時,該種蛋白增多達異常程度,並在大腦神經元內,產生神經纖維纏結。相較於健康大腦,哪些阿茲海默症患者,其大腦功能遭到了,此些最終導致認知損傷之纏結的擾亂。

 

The latest study was proven to be well-tolerated by the participants, all of whom completed the treatment period and over 90% completed the post-treatment periods.

該項最近的研究證實,參與者耐受性良好。全部完成了治療期,且超過90%完成了治療後期。

 

"The results are a significant step forward in demonstrating that we can successfully target tau with a gene silencing drug to slow - or possibly even reverse - Alzheimer's disease, and other diseases caused by tau accumulation in the future," said Dr Catherine Mummery, consultant neurologist at the National Hospital for Neurology and Neurosurgery.

英國國家神經病學暨神經外科醫院神經科顧問醫生,Catherine Mummery博士宣稱:「這些結果是邁向證實,於未來我們能使用一種靜默藥物,成功鎖定tau來減緩,甚至可能逆轉阿茲海默症,及因tau積累引起之其他疾病的重大一步。」

 

While speaking to the Telegraph, Tara Spires-Jones, professor of neurodegeneration and deputy director of the Centre for Discovery Brain Sciences at the University of Edinburgh, showered praises on the findings. Spires-Jones, however, noted there was still a long way to go with the research.

儘管英國愛丁堡大學神經退化學教授,兼發現腦科學中心副主任,Tara Spires-Jones向《每日電訊報》表示,對此些研究結果大加讚揚。不過,Spires-Jones特別提及,關於該項研究仍有漫長的路要走。

 

"Excitingly, in this small study, there was also a very promising reduction of tau in the cerebrospinal fluid after treatment. While there is a long way to go in larger trials to determine whether this drug will help people living with dementia, the data are very promising.

「令人興奮的是,在該項小規模研究中,於治療後腦脊髓液中之tau蛋白,也有非常有指望的減少。儘管在更大規模的試驗中,要確定這種藥物是否,能有助於癡呆症患,有漫長的路要走。不過,此些數據是極有指望的。

 

This type of treatment targeting tau has the potential to slow or even hopefully stop Alzheimer's disease progression, so I very much look forward to seeing results from the next stages of testing."

這種鎖定tau蛋白的療法具有,減緩或甚至有望,阻止阿茲海默症進展的潛力。因此,我非常期待,看到來自下一階段試驗的結果。」

 

 

1. 該項突破可能引領出,癡呆症及阿茲海默症的諸多新療法。

This breakthrough could lead to new treatments for dementia and Alzheimer's disease. 

 

 

網址:https://www.medicaldaily.com/gene-silencing-technology-offers-fresh-hope-alzheimers-patients-469575

翻譯:許東榮

台長: peregrine
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