A new mouse model of an inherited form of dystonia has shown the spinal cord is the driver of the condition, overturning previous understanding that the movement disorder is caused by disruption of neural circuits in the brain. The connection was demonstrated by selectively deleting torsin family 1 member A (TOR1A), the gene that causes dystonia, in the neurons of the spinal cord only.
The aqueous supernatants resulting from ultracentrifugation of brain samples from patients with Alzheimer’s disease (AD) contain aggregates so far described as soluble oligomers of amyloid-β protein (Aβ), which are responsible for the neurotoxicity underlying AD and thus considered targets to watch in this devastating condition. Now, a group of scientists from Harvard Medical School have determined that these aggregates are in fact insoluble diffusible fibrils with the same atomic structure as plaque fibrils.
The human genome, the sequence that represents the DNA of our species, was built with a single individual as a model. This all-in-one standard didn’t include the gene variations that make us different or explain why some people develop certain diseases. Four simultaneous studies from the Human Pangenome Reference Consortium have published a sequence based on 47 individuals, beginning to capture the genetic diversity that defines humans.
Researchers from Hibercell Inc. presented preclinical data for the eukaryotic translation initiation factor 2-α kinase 3 (PERK) inhibitor HC-5404, currently in phase I development for the treatment of solid tumors (NCT04834778).
Pyrimidopyridine derivatives acting as GTPase KRAS G12D inhibitors have been reported in a Sunshine Lake Pharma Co. Ltd. patent as potentially useful for the treatment of cancer.
Beijing Tide Pharmaceutical Co. Ltd. has prepared proteolysis-targeting chimera (PROTAC) compounds comprising an ubiquitin ligase binding moiety bound to a protein phosphatase non-receptor type 11 (PTPN11; PTP-2C; SHP-2) targeting moiety through a linker.
Research at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. has led to the identification of sulfonylpiperazinyl compounds acting as UDP-2,3-diacylglucosamine hydrolase (LpxH) inhibitors and reported to be useful for the treatment of gram-negative bacterial infections.
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