Primary angle closure glaucoma (PACG) is characterized by anatomic blockage of the drainage angle of the eye leading to elevated eye pressure and optic neuropathy, and its molecular pathogenesis is still not well studied.
Small nuclear RNAs (snRNAs) play a crucial role in RNA splicing and cell functioning. The transcription of these RNAs is initiated by small nuclear RNA activation protein complex (SNAPc), and SNAPC4 is the subunit in charge of SNAPc-DNA binding. Previous studies have found that SNAPC4 deficiency led to decreased expression of these RNAs in animal models.
Investigators from 35Pharma Inc. carried out in vivo testing of HS-135, an activin-receptor-based fusion protein (ActRIIB ligand trap), focusing on target engagement, effect on body composition and efficacy in pulmonary arterial hypertension (PAH).
Researchers from Children's Hospital Los Angeles presented data from a study that linked a homozygous missense mutation in ARSK to a new subtype of the lysosomal storage disease mucopolysaccharidoses (MPS).
G protein-coupled receptor 183 (GPR183/EBI2) is one of the key regulators of the innate immune system, and it has been shown to be upregulated in multiple sclerosis (MS) plaques. In a recently reported study, researchers from Medical University of Gdansk evaluated the effects of pharmacological modulation of GPR183 signaling on remyelination.
Promidis Srl has presented results from preclinical evaluations of potent capsid assembly modulators (CAMs) to prevent abnormal accumulation of empty capsids and associated T-cell-mediated liver injury.