A gene therapy for inherited blindness using dCas9-VPR–mediated transcriptional activation
Sybille Böhm, Victoria Splith, Lisa Maria Riedmayr, René D. Rötzer, Gilles Gasparoni, Karl Nordström, Johanna Wagner, Klara Sonnie Hinrichsmeyer, Jörn Walter, Christian Wahl‐Schott, Stefanie Fenske, Martin Biel, Stylianos Michalakis, Elvir Bećirović
Abstract
) in a rhodopsin-deficient mouse model for retinitis pigmentosa. One year after treatment, this approach yields improved retinal function and attenuated retinal degeneration with no apparent adverse effects. Our study demonstrates that dCas9-VPR-mediated transcriptional activation of functionally equivalent genes has great potential for the treatment of genetic disorders.
Topics & Concepts
BiologyRetinitis pigmentosaGeneTranscriptional regulationRegulation of gene expressionGeneticsRhodopsinCell biologyFunction (biology)Gene expressionComputational biologyRetinalBiochemistryRetinal Development and DisordersCRISPR and Genetic EngineeringVirus-based gene therapy research