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Design, synthesis, and biological evaluation of novel HDAC3 PROTACs for combined therapy with Venetoclax in acute myeloid leukemia.

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Bioorganic chemistry 📖 저널 OA 2.3% 2024: 0/13 OA 2025: 1/75 OA 2026: 4/129 OA 2024~2026 2026 Vol.170() p. 109520
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Chen Y, Liu E, Ding X, Qin C, Jiang Y, Li X

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Acute myeloid leukemia (AML) is a heterogeneous hematopoietic neoplasm driven in part by aberrant epigenetic mechanisms such as histone deacetylation, with HDAC3 playing an important role in its patho

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APA Chen Y, Liu E, et al. (2026). Design, synthesis, and biological evaluation of novel HDAC3 PROTACs for combined therapy with Venetoclax in acute myeloid leukemia.. Bioorganic chemistry, 170, 109520. https://doi.org/10.1016/j.bioorg.2026.109520
MLA Chen Y, et al.. "Design, synthesis, and biological evaluation of novel HDAC3 PROTACs for combined therapy with Venetoclax in acute myeloid leukemia.." Bioorganic chemistry, vol. 170, 2026, pp. 109520.
PMID 41610583 ↗

Abstract

Acute myeloid leukemia (AML) is a heterogeneous hematopoietic neoplasm driven in part by aberrant epigenetic mechanisms such as histone deacetylation, with HDAC3 playing an important role in its pathogenesis. In this study, we designed a series of novel HDAC3 PROTACs based on our previously designed HDAC3 inhibitor as the POI ligand. Among these, representative compound B22 potently degraded HDAC3, with a DC of 30.73 nM and a D of 82%. B22 synergized with the clinically approved AML agent Venetoclax, demonstrating potent anti-proliferative effects and significantly promoting apoptosis in MV4-11 cells. Mechanistically, the combination of B22 and Venetoclax synergistically induces DNA damage and downregulates the anti-apoptotic proteins Mcl-1 and Bcl-xL. In conclusion, this study provided insights into novel HDAC3-directed PROTACs development and proposed their therapeutic potential against AML.

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