DEAD-box helicase 17 (DDX17) is an RNA helicase involved in the early phases of neuronal differentiation. Researchers have identified a total of 13 patients presenting with neurodevelopmental phenotypes and who harbored de novo monoallelic variants in the DDX17 gene. The phenotype was characterized by intellectual disability, delayed speech and language, as well as motor delay.
Genetic alterations in FAT1, YAP1 or WWTR1 genes are commonly seen in head and neck squamous cell carcinoma (HNSCC) patients. Targeting Hippo and MAPK pathways in combination has proven effective in preclinical models of HNSCC.
At the recent SITC meeting in Houston, Teva Pharmaceutical Industries Ltd. reported preclinical data for the fusion protein TEV-56278, which is in phase I development.
The next-generation farnesyl transferase inhibitor KO-2806 is currently in phase I clinical development at Kura Oncology Inc. for the treatment of patients with solid tumors, including non-small-cell lung cancer (NSCLC) and colorectal cancer (CRC).
WDR45 is located on the X chromosome. Its pathogenic variants are associated with various neurodegenerative disorders that are predominantly reported in females and present a wide range of clinical phenotypes, from early-onset developmental delay to neurodegeneration and multiple epileptic syndromes.
CK1α serves as an upstream regulator of the p53 pathway, and its degradation may facilitate cell death by preventing MDM2 and/or MDMX mediated inactivation of p53.
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