Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing
Susie Suh, Elliot H. Choi, Henri Leinonen, Andrzej T. Foik, Gregory A. Newby, Wei-Hsi Yeh, Zhiqian Dong, Philip D. Kiser, David C. Lyon, David R. Liu, Krzysztof Palczewski
Topics & Concepts
RPE65BiologyNonsense mutationPoint mutationRetinalGeneticsIndelTranscription activator-like effector nucleaseBase pairMutationMolecular biologyDNAGeneGenome editingCRISPRGenetic enhancementBiochemistryMissense mutationSingle-nucleotide polymorphismGenotypeCRISPR and Genetic EngineeringMosquito-borne diseases and controlCytomegalovirus and herpesvirus research