分子ピンセットがタンパク質を破壊
(c) UC Regents
UCLAのGal Bitan准教授
アルツハイマー病の原因となるアミロイドβおよびタウと呼ばれるタンパク質の塊を，分子ピンセットCLR01が壊すことが動物実験で明らかになった。CLR01は最近，パーキンソン病に関与するタンパク質の蓄積を防ぐ分子化合物として開発された。C字型をしており，タンパク質に含まれるリジンというアミノ酸を包み込んで結合する性質を持つ。この実験で，CLR01は血液‐脳関門を安全に通過し，シグナル伝達に重要な神経細胞間の接合部分，シナプスを保護する効果も示した。毒性の徴候もなく，アルツハイマー病やパーキンソン病の治療法としての開発に有望と考えられる。（吉田素子）
Researchers Report Potential New Treatment to Stop Alzheimer's Disease
Molecular 'Tweezers' Break up Toxic Aggregations of Proteins in Mouse Model
Last March, researchers at UCLA reported the development of a molecular compound called CLR01 that prevented toxic proteins associated with Parkinson's disease from binding together and killing the brain's neurons.
Building on those findings, they have now turned their attention to Alzheimer's disease, which is thought to be caused by a similar toxic aggregation or clumping, but with different proteins, especially amyloid-beta and tau.
And what they've found is encouraging. Using the same compound, which they've dubbed a "molecular tweezer," in a living mouse model of Alzheimer's, the researchers demonstrated for the first time that the compound safely crossed the blood-brain barrier, cleared the existing amyloid-beta and tau aggregates, and also proved to be protective to the neurons' synapses -- another target of the disease -- which allow cells to communicate with one another.
The report appears in the current online edition of the journal Brain.
"This is the first demonstration that molecular tweezers work in a mammalian animal model," said Gal Bitan, an associate professor of neurology at UCLA and the senior author of the study. "Importantly, no signs of toxicity were observed in the treated mice. The efficacy and toxicity results support the mechanism of this molecular tweezer and suggest these are promising compounds for developing disease-modifying therapies for Alzheimer's disease, Parkinson's and other disorders."
Molecular tweezers are complex molecular compounds capable of binding to other proteins.
［単語和訳］
Researchers：研究者 Alzheimer's Disease：アルツハイマー病 Molecular：分子の Tweezers：ピンセット Break up：～を砕く Toxic：有毒な Aggregations：集合体，凝集 Proteins：タンパク質 Mouse：マウス
compound：化合物 associated with：～に関連した Parkinson's disease：パーキンソン病 binding ：結合 brain's：脳の neurons：ニューロン
Building on：～に基づいて have turned their attention to：今度は～に注目した or ：すなわち clumping：凝集 amyloid-beta：アミロイドβタンパク質 tau：タウタンパク質
encouraging：後押しする，励みになる dubbed：～と呼んだ crossed ：通過した blood-brain barrier：血液脳関門 aggregates：凝集物 proved to be：～であることが判明した protective to：～を保護する synapses：シナプス cells：細胞
online edition：オンライン版
demonstration：実証 mammalian：哺乳類の associate professor：准教授 neurology：神経学 senior author：主席著者 Importantly：重要なことには toxicity：毒性 treated：処置された efficacy：有効性 disease-modifying therapies：病態修飾療法
