cereblon 是来那度胺和泊马度胺免疫调节和抗增殖活性的直接蛋白靶标。

Cereblon is a direct protein target for immunomodulatory and antiproliferative activities of lenalidomide and pomalidomide.

机构信息

Celgene, San Diego, CA 92121, USA.

出版信息

Leukemia. 2012 Nov;26(11):2326-35. doi: 10.1038/leu.2012.119. Epub 2012 May 3.

Abstract

Thalidomide and the immunomodulatory drug, lenalidomide, are therapeutically active in hematological malignancies. The ubiquitously expressed E3 ligase protein cereblon (CRBN) has been identified as the primary teratogenic target of thalidomide. Our studies demonstrate that thalidomide, lenalidomide and another immunomodulatory drug, pomalidomide, bound endogenous CRBN and recombinant CRBN-DNA damage binding protein-1 (DDB1) complexes. CRBN mediated antiproliferative activities of lenalidomide and pomalidomide in myeloma cells, as well as lenalidomide- and pomalidomide-induced cytokine production in T cells. Lenalidomide and pomalidomide inhibited autoubiquitination of CRBN in HEK293T cells expressing thalidomide-binding competent wild-type CRBN, but not thalidomide-binding defective CRBN(YW/AA). Overexpression of CRBN wild-type protein, but not CRBN(YW/AA) mutant protein, in KMS12 myeloma cells, amplified pomalidomide-mediated reductions in c-myc and IRF4 expression and increases in p21(WAF-1) expression. Long-term selection for lenalidomide resistance in H929 myeloma cell lines was accompanied by a reduction in CRBN, while in DF15R myeloma cells resistant to both pomalidomide and lenalidomide, CRBN protein was undetectable. Our biophysical, biochemical and gene silencing studies show that CRBN is a proximate, therapeutically important molecular target of lenalidomide and pomalidomide.

摘要

沙利度胺和免疫调节药物来那度胺在血液系统恶性肿瘤中具有治疗活性。普遍表达的 E3 连接酶蛋白 cereblon(CRBN)已被确定为沙利度胺的主要致畸靶标。我们的研究表明,沙利度胺、来那度胺和另一种免疫调节药物泊马度胺与内源性 CRBN 和重组 CRBN-DNA 损伤结合蛋白 1(DDB1)复合物结合。CRBN 介导来那度胺和泊马度胺在骨髓瘤细胞中的抗增殖活性,以及来那度胺和泊马度胺诱导 T 细胞中的细胞因子产生。来那度胺和泊马度胺抑制表达结合沙利度胺能力野生型 CRBN 的 HEK293T 细胞中 CRBN 的自泛素化,但不抑制结合沙利度胺有缺陷的 CRBN(YW/AA)。在 KMS12 骨髓瘤细胞中过表达 CRBN 野生型蛋白,但不过表达 CRBN(YW/AA)突变蛋白,可放大泊马度胺介导的 c-myc 和 IRF4 表达降低以及 p21(WAF-1)表达增加。在 H929 骨髓瘤细胞系中进行来那度胺耐药的长期选择伴随着 CRBN 的减少,而在对泊马度胺和来那度胺均耐药的 DF15R 骨髓瘤细胞中,CRBN 蛋白无法检测到。我们的生物物理、生化和基因沉默研究表明,CRBN 是来那度胺和泊马度胺的近位、治疗上重要的分子靶标。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5af7/3496085/f9339bb8ba57/leu2012119f1.jpg

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