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蛋白酶体抑制用于治疗利什曼病、恰加斯病和昏睡病。

Proteasome inhibition for treatment of leishmaniasis, Chagas disease and sleeping sickness.

作者信息

Khare Shilpi, Nagle Advait S, Biggart Agnes, Lai Yin H, Liang Fang, Davis Lauren C, Barnes S Whitney, Mathison Casey J N, Myburgh Elmarie, Gao Mu-Yun, Gillespie J Robert, Liu Xianzhong, Tan Jocelyn L, Stinson Monique, Rivera Ianne C, Ballard Jaime, Yeh Vince, Groessl Todd, Federe Glenn, Koh Hazel X Y, Venable John D, Bursulaya Badry, Shapiro Michael, Mishra Pranab K, Spraggon Glen, Brock Ansgar, Mottram Jeremy C, Buckner Frederick S, Rao Srinivasa P S, Wen Ben G, Walker John R, Tuntland Tove, Molteni Valentina, Glynne Richard J, Supek Frantisek

机构信息

Genomics Institute of the Novartis Research Foundation, 10675 John Jay Hopkins Drive, San Diego, California 92121, USA.

Wellcome Trust Centre for Molecular Parasitology, Institute of Infection, Immunity and Inflammation, College of Medical, Veterinary and Life Sciences, University of Glasgow, Glasgow G12 8TA, UK.

出版信息

Nature. 2016 Sep 8;537(7619):229-233. doi: 10.1038/nature19339. Epub 2016 Aug 8.

Abstract

Chagas disease, leishmaniasis and sleeping sickness affect 20 million people worldwide and lead to more than 50,000 deaths annually. The diseases are caused by infection with the kinetoplastid parasites Trypanosoma cruzi, Leishmania spp. and Trypanosoma brucei spp., respectively. These parasites have similar biology and genomic sequence, suggesting that all three diseases could be cured with drugs that modulate the activity of a conserved parasite target. However, no such molecular targets or broad spectrum drugs have been identified to date. Here we describe a selective inhibitor of the kinetoplastid proteasome (GNF6702) with unprecedented in vivo efficacy, which cleared parasites from mice in all three models of infection. GNF6702 inhibits the kinetoplastid proteasome through a non-competitive mechanism, does not inhibit the mammalian proteasome or growth of mammalian cells, and is well-tolerated in mice. Our data provide genetic and chemical validation of the parasite proteasome as a promising therapeutic target for treatment of kinetoplastid infections, and underscore the possibility of developing a single class of drugs for these neglected diseases.

摘要

恰加斯病、利什曼病和昏睡病影响着全球2000万人,每年导致超过5万人死亡。这些疾病分别由动基体寄生虫克氏锥虫、利什曼原虫属和布氏锥虫属感染引起。这些寄生虫具有相似的生物学特性和基因组序列,这表明所有这三种疾病都可以用调节保守寄生虫靶点活性的药物治愈。然而,迄今为止尚未发现此类分子靶点或广谱药物。在此,我们描述了一种对动基体蛋白酶体具有选择性的抑制剂(GNF6702),其在体内具有前所未有的疗效,在所有三种感染模型中均能从小鼠体内清除寄生虫。GNF6702通过非竞争性机制抑制动基体蛋白酶体,不抑制哺乳动物蛋白酶体或哺乳动物细胞的生长,并且在小鼠中耐受性良好。我们的数据为寄生虫蛋白酶体作为治疗动基体感染的一个有前景的治疗靶点提供了遗传学和化学验证,并强调了为这些被忽视的疾病开发单一类药物的可能性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5b12/5161665/9fc3ed81c240/nihms807671f5.jpg

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