Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa

M. Dominik Fischer, Michelle E. McClements, Cristina Martinez-Fernandez de la Camara, Julia Sophia Bellingrath, Daniyar Dauletbekov, Simon C. Ramsden, Doron G. Hickey, Alun R. Barnard, Robert E MacLaren

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Biochemistry, Genetics and Molecular Biology