Bart Klein was at the forefront of ProQR's Axiomer® RNA editing technology development. This platform technology makes it possible to use the cells own machinery to reverse mutations that cause a genetic disease. Read the interview.
ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.
… a rare genetic disorder, with the objective to develop RNA therapies for rare diseases. Prior to founding ProQR, … In 1992 Dr. Valerio was appointed professor of gene therapy at the University of Leiden, where he also received …
… Always excited about RNA therapies He is one of the founders of ProQR, but even … as they say. Gerard Platenburg What made you pursue RNAtherapy as a potential treatment for these diseases? “Well, …
2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.
This month several ProQRians will attend the Oligonucleotide and Peptide Therapeutics Conference (TIDES Europe) 2022 to highlight our Axiomer® RNA-editing platform technology.
