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Engineered Polymersomes for the Treatment of Fish Odor Syndrome: A First Randomized Double Blind Olfactory Study.
Advanced Science ( IF 15.1 ) Pub Date : 2020-03-09 , DOI: 10.1002/advs.201903697
Aaron C Schmidt 1 , Erik R Hebels 1 , Charlotte Weitzel 1 , Anna Kletzmayr 1 , Yinyin Bao 1 , Christian Steuer 1 , Jean-Christophe Leroux 1
Affiliation  

Trimethylamine (TMA) is a metabolite overtly present in patients suffering from trimethylaminuria (TMAU), a rare genetic disorder characterized by a strong "fishy" body odor. To date, no approved pharmacological treatment to sequester excess TMA on the skin of patients exists. Here, transmembrane pH gradient poly(isoprene)-block-poly(ethylene glycol) (PI-b-PEG) polymersomes are investigated for the topical removal of TMA. PI-b-PEG amphiphiles of varying chain length are synthesized and evaluated for their ability to form vesicular structures in aqueous media. The optimization of the PI/PEG ratio of transmembrane pH gradient polymersomes allows for the rapid and efficient capture of TMA both in solution and after incorporation into a topical hydrogel matrix at the pH of the skin. A subsequent double blind olfactory study reveals a significant decrease in perceived odor intensity after application of the polymersome-based formulation on artificial skin substrates that has been incubated in TMA-containing medium. This simple and novel approach has the potential to ease the burden of people suffering from TMAU.

中文翻译:

用于治疗鱼臭综合症的工程聚合物体:第一项随机双盲嗅觉研究。

三甲胺 (TMA) 是一种明显存在于三甲胺尿症 (TMAU) 患者体内的代谢物,三甲胺尿症是一种罕见的遗传性疾病,其特征是强烈的“鱼腥味”体味。迄今为止,尚无批准的药物治疗方法可以将过量的 TMA 隔离在患者皮肤上。在这里,研究了跨膜 pH 梯度聚(异戊二烯)-嵌段-聚(乙二醇)(PI-b-PEG)聚合物囊泡的局部去除 TMA。合成了不同链长的 PI-b-PEG 两亲物,并评估了它们在水介质中形成囊泡结构的能力。跨膜 pH 梯度聚合物囊泡的 PI/PEG 比例的优化可以在溶液中以及在皮肤 pH 值下掺入局部水凝胶基质后快速有效地捕获 TMA。随后的双盲嗅觉研究表明,在含有 TMA 的介质中孵育的人造皮肤基材上应用基于聚合物囊泡的配方后,感知到的气味强度显着降低。这种简单而新颖的方法有可能减轻 TMAU 患者的负担。
更新日期:2020-04-21
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