Reduction of renal tubular cell polynucleotide phosphorylase (PNPT1) expression has been linked to renal tubular atrophy in chronic kidney disease, according to a new study. The research, published in Nature Communications, found that renal tubular cell PNPT1 reduction causes renal tubular atrophy by inhibiting protein synthesis.

Researchers from Onxeo SA presented preclinical data for OX-425, a first-in-class oligodeoxynucleotide that operates as a poly (ADP-ribose) polymerase 1 (PARP-1) decoy, and which is being developed as anticancer agent.

Homozygous familial hypercholesterolemia (HoFH) is a severe rare life-threatening condition where high blood levels (>500 mg/dL) of low-density lipoprotein cholesterol (LDL-C), triglycerides (TG), and premature and progressive atherosclerotic cardiovascular disease (ASCVD) are the main features.

Molecular Templates Inc. has received IND clearance from the FDA for its novel MT-8421 engineered toxin bodies (ETB) program targeting cytotoxic T-lymphocyte protein 4 (CTLA-4) in patients with relapsed/refractory solid tumors previously exposed to checkpoint inhibitors. MT-8421 is designed to eliminate CTLA-4-expressing regulatory T cells (Tregs) in the tumor microenvironment (TME) through a direct cell-kill mechanism independent of the effector cell presence that antibodies rely upon while not affecting Tregs in the periphery.

Researchers from Wenzhou Medical University and affiliated organizations presented the discovery of novel anti-inflammatory agents. Synthesis and optimization of a series of 4-oxo-N-phenyl-1,4-dihydroquinoline-3-carboxamide derivatives led to the identification of compound [I] as the lead, as it exhibited the best inhibitory effect in vitro. More specifically, [I] demonstrated the strongest inhibitory effect on LPS-induced TNF-α (82.58%) and IL-6 (75.05%) expression, while exhibiting no cytotoxicity at 10 μM in J774A.1 macrophages.

A deficiency in fumarate metabolism could be behind a new mechanism of inflammation mediated by mitochondrial DNA and RNA. Two independent and simultaneous studies described how the accumulation of fumarate in the mitochondria released the genetic material of this organelle through vesicles, activating an inflammatory signaling pathway.

The intended use of gene editing tools on pre-implantation human embryos would be to avoid the development of congenital diseases in the upcoming baby. But it may have its own risks. Those risks were illustrated in a publication in the March 7, 2023, issue of Nature Communications, where researchers from the Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University (OHSU) showed that the method that is most frequently used for evaluating the effects of gene editing zygotes did not always result in an accurate picture of those edits.