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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).
Sirna Therapeutics, Inc. was a San Francisco, California based biotechnology company that explored the use of RNA interference in human disease therapy. Sirna's development pipeline included several small interfering RNA (siRNA) drugs , thought to stably silence the expression of specific disease-related genes.
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 ...
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.
The second frame codes for an RNA-dependent RNA polymerase (and its helper proteins) which replicates the mRNA construct in the cell. This allows smaller vaccine doses. [ 100 ] The mechanisms and consequently the evaluation of self-amplifying mRNA may be different, as self-amplifying mRNA is a much bigger molecule.
Cancer and Genetics Research Complex [1] is an interdisciplinary research center located at the University of Florida (UF). The goal of this facility is "to harness the diverse academic talents and resources of the genetic research community at UF to improve the health and well-being of our citizenry."
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