Bietti’s crystalline corneoretinal dystrophy (BCD) is an autosomal recessive inherited disease caused by mutations in the cytochrome P450 (CYP) family 4 subfamily V member 2 (CYP4V2) gene, which encodes a polyunsaturated fatty acid (PUFA) hydroxylase dominantly expressed in retinal pigment epithelium (RPE) cells.
Mutations in the RPGRIP1 gene are associated with rare retinal dystrophies and most commonly with Leber congenital amaurosis (LCA) type 6, which is characterized by vision loss, among other symptoms.
Intron Biotechnology Inc. has announced the identification of lysogenic bacteriophages prophage and jamphage in the pancreatic cancer-related microbiome. This identification was achieved as part of the ongoing Phageriarus development project that is focused on acquiring bacteriophage-derived proteins that can serve as immune regulators, with the ultimate goal of developing phage-based immunotherapeutics for immune disorders and cancer.
Glioblastoma multiforme (GBM) is an aggressive brain cancer with poor prognosis and survival. TNF-related apoptosis-inducing ligand (TRAIL) is a protein that induces apoptosis in cancer cells by binding death receptors type 4 and 5. Researchers at the University of North Carolina explored using hiNeuroS-TRAIL combined with the ClpP activator TR-107 as a potential treatment.
Hilleman Laboratories Singapore Pte Ltd. and the Agency for Science, Technology and Research (A*STAR) have established a collaboration to explore using novel circular ribonucleic acid (circRNA) technology to develop a Nipah virus vaccine and to validate the technology platform for application for other infectious diseases pathogens.
At the recent ATS meeting, Tavanta Therapeutics Inc. introduced TAVT-135, a cell-penetrating peptide (CPP) conjugate that acts as a chloride ion (Cl-) transporter. The product is currently being investigated for cystic fibrosis (CF), regardless of CF transmembrane conductance regulatory (CFTR) gene mutation status.
Idiopathic pulmonary fibrosis (IPF) is a rare, progressive and chronic lung disease that causes scarring of the lung tissue and leads to an irreversible decline in lung function. Several studies have linked the p53 tumor suppressor gene to pulmonary fibrosis.
Researchers have discovered that a subunit of the ubiquitin-proteasome system acted independently of the proteasome machinery to regulate AMPA receptors, a type of glutamate receptor, at multiple steps of their life cycle. Published in the May 26, 2023, issue of Science, the findings could point to ways to target AMPA receptors. They are responsible for the majority of excitatory transmission in the central nervous system, and current drugs seeking to influence AMPA-based transmission are “good but they are not great,” Erin Schuman told BioWorld. “This regulatory particle is watching the glutamate receptor at each step.” Schuman is the director of the Max Planck Institute for Brain Research and the paper’s senior author.
Shenzhen Winkey Technology Co. Ltd. has described tetrapeptide derivatives reported to be useful for the treatment of skin inflammation and hyperpigmentation.
Medshine Discovery Inc. has divulged azaspiro compounds acting as leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease.
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