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Sorafenib and glimepiride simultaneously downregulated c-Maf proteinexpression to induce G1 phase arrest and apoptosis in myeloma cells. Given the abnormally high expression of c-Maf in most MM patients, targeting c-Maf presents an attractive therapeutic approach for treating MM malignancies.
Other researchers are investigating approaches that use AAV-mediated trophic support and antibody-based therapies, such as vaccines and smallmolecule drugs, to modify various proteinexpressions. For example, antioxidant-based treatments may counter the higher levels of oxidative stress in ALS patients.
These multifunctional smallmolecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.
Recently published literature has identified three proteins (Lap2β, H3K9me3, and HP1γ) that exhibit decreased expression in aged fibroblasts and a “senescence cocktail” of smallmolecules that have been observed to reduce the expression of these chromatin proteins in a way that models aged cells.
Other data to be presented will show high treatment persistence and strong adherence for patients treated with OCREVUS compared to other DMTs in real-world settings. Fenebrutinib is designed to be a highly selective smallmolecule and is the only reversible (non-covalent) BTK inhibitor currently in Phase III development in MS.
They work by binding to specific sequences of nucleotides present within the mRNA structure and can induce mechanisms that either decrease, restore, or modify proteinexpression. And since proteins are often linked to disease, there's huge potential to treat a broad range of diseases with this technology.
Overviews of clinical trials and scientific rationale will be presented at MSVirtual2020, the 8th Joint Meeting of the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) from 11-13 September 2020.
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