近日,，國(guó)際著名雜志The Journal of Experimental Medicine刊登了英國(guó)研究人員的最新研究成果“A novel neuroprotective therapy for Parkinson’s disease using a viral noncoding RNA that protects mitochondrial Complex I activity ,。”,，文章中，研究人員探索出一種治療帕金森氏癥的新方法,，這種方法已經(jīng)在動(dòng)物實(shí)驗(yàn)中顯示有效,，如能通過(guò)人類(lèi)臨床試驗(yàn)，將有望用于治療早老性癡呆等其他神經(jīng)系統(tǒng)疾病,。
英國(guó)劍橋大學(xué)研究人員在新一期《實(shí)驗(yàn)醫(yī)學(xué)雜志》上報(bào)告說(shuō),，大腦神經(jīng)細(xì)胞中線(xiàn)粒體的功能異常是導(dǎo)致帕金森氏癥的重要原因，為了保護(hù)線(xiàn)粒體的功能,，他們想到了向病毒“學(xué)習(xí)”,。
病毒雖然是人體的敵人，但它們?cè)谇秩肴梭w細(xì)胞后,，為了有充分時(shí)間利用人體細(xì)胞的資源來(lái)自我復(fù)制,，會(huì)想辦法保護(hù)作為細(xì)胞能量工廠(chǎng)的線(xiàn)粒體的功能，讓線(xiàn)粒體多工作一段時(shí)間,。在這個(gè)過(guò)程中,，指導(dǎo)病毒保護(hù)線(xiàn)粒體的是一個(gè)名為“貝塔2.7”的基因。
于是研究人員想辦法把這個(gè)基因送入患有帕金森氏癥的實(shí)驗(yàn)鼠大腦中,，結(jié)果顯示神經(jīng)細(xì)胞的線(xiàn)粒體也受到保護(hù),，帕金森氏癥得到有效治療。這個(gè)方法的另一個(gè)好處是,，用來(lái)搭載這個(gè)基因的載體是一種特殊的蛋白質(zhì),，它可以通過(guò)血管和大腦神經(jīng)系統(tǒng)之間的邊界，因此這種治療方法只需要進(jìn)行血液注射,，避免了對(duì)大腦進(jìn)行侵入性的治療,。
領(lǐng)導(dǎo)研究的約翰·辛克萊爾教授說(shuō)，研究結(jié)果從原則上證明了這種方法對(duì)帕金森氏癥的有效性,，接下來(lái)將進(jìn)行人類(lèi)臨床試驗(yàn),，以探索治療人類(lèi)患者所需的劑量和頻率等問(wèn)題。
帕金森氏癥是一種常見(jiàn)于中老年人的神經(jīng)系統(tǒng)疾病,，主要癥狀是震顫,、動(dòng)作遲緩、肌肉僵硬等,。（生物谷Bioon.com）
doi:10.1084/jem.20111126
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A novel neuroprotective therapy for Parkinson’s disease using a viral noncoding RNA that protects mitochondrial Complex I activity
Wei-Li Kuan1,2, Emma Poole3, Michael Fletcher4, Sharon Karniely3, Pam Tyers1,2, Mark Wills3, Roger A. Barker1,2, and John H. Sinclair3
Parkinson’s disease (PD) is a neurodegenerative disorder that results in the loss of nigrostriatal dopamine neurons. The etiology of this cell loss is unknown, but it involves abnormalities in mitochondrial function. In this study, we have demonstrated that the administration of a novel noncoding p137 RNA, derived from the human cytomegaloviral β2.7 transcript, can prevent and rescue dopaminergic cell death in vitro and in animal models of PD by protecting mitochondrial Complex I activity. Furthermore, as this p137 RNA is fused to a rabies virus glycoprotein peptide that facilitates delivery of RNA across the blood–brain barrier, such protection can be achieved through a peripheral intravenous administration of this agent after the initiation of a dopaminergic lesion. This approach has major implications for the potential treatment of PD, especially given that this novel agent could have the same protective effect on all diseased neurons affected as part of this disease process, not just the dopaminergic nigrostriatal pathway.
