X-linked sideroblastic anemia (XLSA) is a rare genetic disorder caused by mutations in the ALAS2 gene, which plays a critical role in heme biosynthesis within red blood cells.
Researchers from the Mayo Clinic described the efficacy of NPA-7, a potential first-in-class multivalent fusion protein comprising a sequence of 22 amino acids of human BNP fused to the Mas receptor agonist Ang 1-7.
Repair Biotechnologies Inc. has received encouraging feedback from a pre-IND meeting with the FDA as it works toward an IND filing to conduct a phase Ib study of its REP-0003 mRNA therapy in patients with homozygous familial hypercholesterolemia (HoFH).
A disintegrin and metalloprotease domain-like protein decysin-1 (ADAMDEC1) is a multifunctional transmembrane glycoprotein that has been shown to promote metastasis and development of various tumor types by degrading the extracellular matrix. Scientists at Nankai University and affiliated organizations aimed to assess the regulatory mechanisms of ADAMDEC1 in the highly aggressive cancer cholangiocarcinoma (CCA).
The activation of the adenosine A2A receptor, which is mediated by the inhibition of adenosine A1 receptor, has been associated with depression-like behavior and anhedonia. High levels of cortisol, increased oxidative stress and antioxidant enzyme reduction are also contributors to the pathophysiology of depression.
A paracrine hormone of skeletal muscle acts as an endocrine signal for the synthesis of a sex hormone. Scientists at McGill University have observed in mice how myostatin, which inhibits the development of muscle mass to control its growth, also stimulated the production of the pituitary follicle-stimulating hormone (FSH), modulating ovarian function. The hypothalamus-pituitary-gonadal axis could include an additional element through a yet unknown role in muscles.
Azothbio Inc. and Daegu-Gyeongbuk Medical Innovation Foundation have described non-receptor tyrosine-protein kinase TYK2 inhibitors reported to be useful for the treatment of autoimmune disease, cancer, endocrine, inflammatory and neurological disorders.
The University of Michigan has synthesized proteolysis targeting chimeras (PROTACs) comprising a von Hippel-Lindau disease tumor suppressor (VHL)-binding moiety coupled to a signal transducer and activator of transcription 3 (STAT3)-targeting moiety via a linker.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have divulged triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of amyotrophic lateral sclerosis, Alzheimer’s disease, frontotemporal dementia, multiple sclerosis, Nasu-Hakola disease, Parkinson’s disease, rheumatoid arthritis and stroke.
Northeastern University has disclosed cannabinoid CB1 receptor allosteric modulators and monoglyceride lipase (MGLL; MAGL) inhibitors reported to be useful for the treatment of pain, inflammation, anxiety, psychosis, traumatic brain injury, post-traumatic stress disorder, epilepsy and neurodegenerative diseases.
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