Article
Locking CBL TKBD in its native conformation presents a novel therapeutic opportunity in mutant CBL-dependent leukemia.
Molecular therapy : the journal of the American Society of Gene Therapy - 6 Aug 2025
Ahmed Syed Feroj, Anand Jayanthi, Zhang Wei, Buetow Lori, Rishi Loveena, Mitchell Louise, Bohlen Jonathan, Lilla Sergio, Sibbet Gary J, Nixon Colin, Patel Amrita, Majorek Karolina A, Zanivan Sara, Bustamante Jacinta C, Sidhu Sachdev S, Blyth Karen, Huang Danny T
Abstract excerpt
Casitas B-lineage lymphoma (CBL) is an E3 ubiquitin ligase critical for negatively regulating receptor protein tyrosine kinases (RTKs). Deleterious CBL mutants lose E3 activity, but act as adaptors that gain function to cause myeloproliferative neoplasms. Currently, there is no targeted treatment available for patients with CBL mutant-dependent disorders. By combining phage-display technology and structure-based...
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