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RNA therapeutics are a new class of medications based on ribonucleic acid (RNA). Research has been working on clinical use since the 1990s, with significant success in cancer therapy in the early 2010s. [1] In 2020 and 2021, mRNA vaccines have been developed globally for use in combating the coronavirus disease (COVID-19 pandemic). [2]
Recent discoveries implicating RNA in the pathogenesis of several forms of cancer and neuromuscular diseases have created a paradigm shift in drug discovery.This work combined with advances in structural characterization techniques such as NMR spectroscopy, X-ray crystallography, Cryogenic electron microscopy [1] together with computational modeling, [2] has pushed forward the realization that ...
There are many additional therapeutics that have been developed and are either in phase I or II of the clinical trials. Current RNA therapeutics in clinical trials range from a variety of target organs and diseases ranging from skin (potential treatment for disease such as keloid) to tumors (squamous cell lung cancer).
Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. [1]
The company develops RNA therapeutics for the treatment of rare diseases such as ornithine transcarbamylase deficiency, and respiratory diseases such as cystic fibrosis. As of 2021 [update] , vaccine medicines include a vaccine candidate for COVID-19 and an influenza vaccine in the preclinical development phase.
Oklahoma State University Center for Health Sciences (OSU-CHS) is a public medical school in Tulsa, Oklahoma. It also has a branch campus in Tahlequah, Oklahoma. Founded in 1972, OSU-CHS is part of the Oklahoma State University System. [5] OSU-CHS offers a Doctor of Osteopathic Medicine (D.O.) and over fifteen other different graduate degrees.
ProQR Therapeutics NV (NASDAQ: PRQR) is a Dutch biotechnology company based in Leiden, the Netherlands, with a presence in Cambridge, Massachusetts, US. [1] The company was funded in 2012 by chief executive officer (CEO) Daniel A. de Boer. It specializes in the development of RNA therapeutics using its RNA editing platform technology called ...
Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company focused on the development of first-in-class drugs that target microRNAs to treat a broad range of diseases. Regulus was established in September 2007 by Alnylam Pharmaceuticals and Isis Pharmaceuticals.