Usher syndrome is the most common cause of deaf-blindness. Mutations in the protocadherin-15 (PCDH15) gene cause Usher syndrome type 1 F (USH1F), which makes up about 3-11% of all Usher syndrome cases. As a component of tip links of the inner ear hair cells, PCDH15 binds to cadherin-23 (CDH23) to convey force from sound stimuli to the mechanosensory transduction channels.
Huchembio Co. Ltd. has described signal transducer and activator of transcription 3 (STAT3) inhibitors reported to be useful for the treatment of autoimmune diseases, atherosclerosis, inflammatory disorders, cancer, diabetic retinopathy, diabetes, osteoporosis and viral infections.
Researchers at Academia Sinica, CSIC (Consejo Superior de Investigaciones Cientificas) and Universidad de Sevilla have divulged 5-aminohexahydro-6,7,8-trihydroxy-3h-oxazolo[3,4-a]pyridin-3-one derivatives acting as Toll-like receptor 4 (TLR4; CD284) antagonists or TLR4 agonists reported to be useful for the treatment of autoimmune disease, metabolic syndrome, asthma, inflammatory bowel disease, allergic rhinitis, nonalcoholic steatohepatitis, atherosclerosis and infections, among others, and also as vaccine adjuvants.
Diamond Therapeutics Inc. has identified deuterated lisuride analogues acting as 5-HT2A receptor agonists reported to be useful for the treatment of neurological disorders.
Currently, there is no FDA-approved drug for nonalcoholic steatohepatitis (NASH), which has evolved into the second leading cause of liver transplantation in the U.S. Researchers from Children’s Hospital Los Angeles and Epigen Biosciences Inc. disclosed preclinical data on EPGN-2154, a novel lysophosphatidic acid LPA1 receptor agonist that has already demonstrated antifibrotic activity in preclinical kidney and liver models.
Dihydroorotate dehydrogenase (DHODH) is an enzyme ubiquitously expressed in all tissues whose inhibition has proven effective in preventing viral replication. Previous knowledge on DHODH inhibitor vidofludimus calcium demonstrated that this compound inhibits viral replication of DNA, RNA and retroviruses and induces innate immune responses that lead to an antiviral state thus inhibiting virus reactivation.
Gastric cancer persists as the fourth leading cause of cancer-related deaths. Syndecans (SDCs) are a family of four transmembrane heparan sulfate proteoglycans involved in cell proliferation, migration and adhesion, among others, and syndecan-4 (SDC4) expression has been shown to be up-regulated during the early stages of the gastric carcinogenesis.
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