The discovery of DNA was a milestone in the history of science that led to a breakthrough in biomedical research. By associating disease and genetics, genome correction techniques were ultimately developed that are supposed to work in the same way that antibiotics and antivirals block pathogenic microorganisms: by directly attacking the causes of disease.
New and updated preclinical data presented at the American Society of Gene & Cell Therapy Congress in Los Angeles, by: 2seventy Bio, Dyne Therapeutics, In8bio, Kelonia Therapeutics, Siren Biotechnology, Umoja Biopharma.
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.
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