Two approaches using an adenine base editor & a Cas9 nuclease prevented the development of hypertrophic cardiomyopathy in mice carrying a pathogenic mutation on the Myh6 gene. GeneticTherapies CardiacDisease
Overhang nucleotides that mediate proper annealing into the sgRNA expression cassette are bolded. Plasmids were purified in high yield and purity by Aldevron for AAV generation.High-throughput sequencing of gDNA was performed as previously describedmMYH6F: 5′-ACACTCTTTCCCTACACGACGCTCTTCCGATCTNNNNCGTGTCACCATCCAGTTGAAC -3′Underlined sequences represent adapters for Illumina sequencing. Primers used to amplify off-target loci are provided in Supplementary Table.
-seq was performed as previously described
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