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.
Researchers have used cell culture experiments to understand how gene expression was affected in a patient with a rare pain insensitivity syndrome, and have identified a network of hundreds of genes whose expression was changed compared to sex-matched controls. Published online in the journal Brain on May 23, 2023, the research is one step toward translating a rare mutation into medications that could provide benefits for common ailments.
Could long non-coding RNAs be the key to developing organ-specific antifibrotic drugs that only mediate their effects in disease-related contexts? That’s the intriguing hypothesis that Haya Therapeutics SA has set out to explore, and its lead program, in heart failure caused by non-obstructive hypertrophic cardiomyopathy, is now in IND-enabling studies. A first clinical trial is pencilled in for late 2024 or early 2025.
Scientists from the Icahn School of Medicine at Mount Sinai have found a sexual dimorphism of depression based on the different expression of a molecule that could be developed as a therapeutic strategy. “There is a big sex difference in depression. Women are much more likely to have depression than men. They tend to have different subsets of symptoms. They tend to respond better to different antidepressants, and the depression tends to be more severe,” Orna Issler, the first author of the study and a postdoctoral researcher at the Nash Family Department of Neuroscience, Friedman Brain Institute, Icahn School of Medicine at Mount Sinai, told BioWorld. Their project, directed by Eric Nestler, a professor of neuroscience and director of the Friedman Brain Institute at the Icahn School of Medicine at Mount Sinai, had the aim to understand the biology of these sex differences of depression and to find therapeutic targets for it.
New research points a finger at a previously unknown long noncoding RNA (lncRNA) as playing a critical role in activating nuclear factor kappa B (NF-kappaB) inflammatory responses and driving the progression of osteoarthritis of the knee.
Sometimes highly impactful serendipitous discoveries are made when performing genetic loss-of-function studies that were initially focused on putative tumor suppressors or other hypotheses.
A long noncoding RNA (lncRNA), the RNA component of mitochondrial RNA-processing endoribonuclease, was shown to promote the growth and proliferation of colorectal cancer cells by inhibiting activity of the tumor suppressor protein p53 in a Chinese study led by oncologists Fudan University Shanghai Cancer Center.
