Dissecting Structural Requirements of Leucinostatin A Derivatives for Antiprotozoal Activity and Mammalian Toxicity.

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Tác giả: Michael Adams, Noëlle Arnold, Marcel Kaiser, Michael Kemmler, Sandra Kurth, Pascal Mäser, Dimanthi Pliatsika, Rainer Riedl, Lukas Rimle, Christoph von Ballmoos

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: United States : Journal of medicinal chemistry , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 98813

Lefleuganan, a clinical stage drug candidate for the treatment of cutaneous leishmaniasis, is a synthetic nonapeptide inspired by the natural antimicrobial peptide leucinostatin A, exhibiting excellent antiprotozoal activity in the low nM range. Lefleuganan shows strongly reduced acute toxicity, making it amenable for clinical use. Here, using a broad set of in vivo and in vitro measurements using purified enzymes, we find that leucinostatin A, but not lefleuganan, specifically targets the mitochondrial ATP synthase, inhibiting ATP synthesis by the human, bovine, and yeast enzyme in the nanomolar range. In a structure-activity relationship study covering the chemical space between the two compounds, hydroxyleucine at position 7 in leucinostatin A is identified as the key responsible moiety for specific ATP synthase inhibition and systemic toxicity. Our data suggest that efficient antiprotozoal activity of these class of compounds is mediated by efficient energetic uncoupling of negatively charged membranes.
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