IKAROS protein stability is regulated by its early N-terminal region and C-terminal dimerization domain.

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Tác giả: Gregor Dueckers, Ana Esteve-Sole, Thomas A Fleisher, Agustin A Gil Silva, Lina Igel, Natchanun Klangkalya, Hye Sun Kuehn, Talal Mousallem, Tim Niehues, Julie E Niemela, Seraina Prader, Sergio D Rosenzweig, Benjamin C Stewart-Bates, Jennifer L Stoddard

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: United States : Clinical immunology (Orlando, Fla.) , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 740626

IKAROS, encoded by IKZF1, is a six zinc-finger (ZF) transcription factor integral to lymphocyte development and function. IKZF1 mutations affecting DNA-binding (ZF1-4) and dimerization (ZF5-6) have been extensively reported and result in human disease. Herein, we investigated IKZF1 mutations affecting protein stability. We identified ten individuals in three families carrying IKZF1 mutations mapping either to the pre-ZF1 area (D22N), or the dimerization domain (M494Vfs*86, Y503*) presenting with infections, immune dysregulation and/or lymphoproliferation with incomplete clinical penetrance. IKAROS expression was reduced in all mutation-carrier evaluated. Protein stability was decreased for D22N, V52L (another pre-ZF1 variant reported in COSMIC), Y503* and Del1-116, a laboratory-designed mutant encompassing the pre-ZF1 area. Mutants Y503* and Del1-116 also exhibited other impaired functions. IKAROS N-terminal pre-ZF1 area, encompassing a previously uncharacterized protein stability-associated region (PSAR), is crucial for IKAROS stability. Variants in the IKAROS PSAR leading to decreased protein stability and IKAROS haploinsufficiency seem sufficient to result in immune defects and IKAROS-associated diseases.
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