Rapid Cohort Generation and Analysis of Disease Spectrum of Large Animal Model of Cone Dystrophy.
More than 60% efficiency in swine with GEG Tech vectors
GUCY2Dmut cDNA encodes a mutant guanylate cyclase 2D with two substitutions: 837 D>E, 838 R>S (modified sequence from human genome, NM_000180.3, 3641 pb). This enzyme is guanylate cyclase 2D with dominant negative substitution D837E and R838S. These mutations have been associated in humans with cone dystrophies.
Rapid Cohort Generation and Analysis of Disease Spectrum of Large Animal Model of Cone Dystrophy.
More than 60% efficiency in swine with GEG Tech vectors
