Epigenetic editing approach used the endogenous cellular mechanisms for regulating gene expression, leading to durable modulation of transcription without affecting the DNA sequence. Chroma Medicine Inc. scientists recently presented a novel epigenetic editing strategy for the treatment of chronic hepatitis B virus (HBV) infection.
Current therapies based on immune checkpoint blockade are effective and offer a valid option for treatment, but many patients develop either primary or acquired resistance to treatment. Previous research has shown that the deletion of protein tyrosine phosphatases PTPN2 and PTPN1 results in an increase in the sensitization of tumor cells and the promotion of antitumor immunity.
Dominantly inherited mutations in the MAPT gene, which encodes the tau protein, result in a subset of tauopathies named frontotemporal lobar degeneration with tau pathology (FTLD-tau). However, the mechanisms by which MAPT mutations cause disease remain unclear. In a recent study, researchers from Washington University in St. Louis aimed to investigate the role of long non-coding RNAs (lncRNAs) and the impact of MAPT mutations on lncRNA in tauopathy.
Protein-arginine deiminase type-4 (PAD4) protein contributes to the formation of neutrophil extranuclear traps (NETs), which in turn lead to tumor growth and cancer immune escape that favors metastatic disease. The expression of PAD4 in certain types of cells, such as hematopoietic stem cells, makes the strategy of PAD4 inhibition risky due to adverse side effects.
Küleon LLC (formerly Psilosterics LLC) has announced a trifunctional serotonergic small molecule that is a full 5-HT2C receptor agonist and full antagonist of the 5-HT2A and 5-HT2B receptors with potential to treat neuropsychiatric disorders. Designated KB-128, it could be developed to treat disorders that can be modulated though 5-HT2C receptors, including schizophrenia, Alzheimer’s psychosis, depression, obesity and addiction.
Just one week after birth, the heart experiences a change in metabolism that helps it meet the high energy demand necessary to fulfill its function. This evolutionary developmental process could have medical advantages.
Recently, researchers at Cincinnati Children’s Hospital, in collaboration with colleagues in Japan, have developed a human vascular organoid model that accurately mimics the vascular damage caused by SARS-CoV-2.
Additional early-stage research and drug discovery news in brief, from: Alligator Bioscience, Athira Pharma, Coya Therapeutics, Kineta, Neurosense Therapeutics, Olatec Therapeutic, Polypid, Sola Biosciences.