Alternative splicing aids in maintaining the complexity of the transcriptome in higher organisms, and small nuclear RNAs (snRNAs), which are major components of the spliceosome, are processed by the integrator complex. Dysfunctions of integrator complex lead to immature snRNA production and may result in secondary defects in mRNA splicing.
Primary biliary cholangitis (PBC) is the most common autoimmune liver disease; several genome-wide association studies have suggested chromosome locus 19p13.3 is associated with PBC pathology. Chinese researchers conducted a case-control study to unveil genetic variants in the 19p13.3 locus associated with PBC. The meta-analysis included 1,931 individuals with PBC and 7,852 controls.
Eli Lilly and Co. has described aryl hydrocarbon receptor (AhR) agonists reported to be useful for the treatment of psoriasis, ulcerative colitis, Crohn’s disease, graft-vs.-host disease and multiple sclerosis.
Medshine Discovery Inc. has divulged halogen-substituted pyridazinone compounds acting as short transient receptor potential channel 5 (TRPC5) antagonists reported to be useful for the treatment of hypertensive nephropathy.
Shanghai Institutes for Biological Sciences and Shanghaitech University have identified dopamine D2 receptor and/or 5-HT receptor agonists reported to be useful for the treatment of depression and schizophrenia.
Black Diamond Therapeutics Inc. has synthesized quinoline derivatives acting as Raf kinase B (B-Raf) mutant inhibitors reported to be useful for the treatment of cancer.
Frontotemporal dementia (FTG-GRN) is a fatal neurodegenerative disease caused by heterozygous mutations in the GRN gene, encoding progranulin (PRGN). Researchers at Arkuda Therapeutics Inc. reported on ARKD-104, a novel potent orally bioavailable PGRN-enhancing compound that increased PRGN secretion and lysosomal granulins in multiple relevant cell lines.