Twin Prime Editing Mediated Exon Skipping/Reinsertion for Restored Collagen VII Expression in Recessive Dystrophic Epidermolysis Bullosa
Benjamin J. Steinbeck, Xin D. Gao, Amber McElroy, Smriti Pandey, Jordan L. Doman, Megan Riddle, Lily Xia, Weili Chen, Cindy Eide, André van Helvoort Lengert, Sang Won Han, Bruce R. Blazar, Hans H. Wandall, Sally Dabelsteen, David R. Liu, Jakub Tolar, Mark J. Osborn
Topics & Concepts
ExonBiologyEpidermolysis bullosaGeneticsGenome editingLocus (genetics)Exon skippingGeneMolecular biologyGenomeAlternative splicingCRISPR and Genetic EngineeringRNA regulation and diseaseAnimal Genetics and Reproduction