Article
Quantitative assessment of timing, efficiency, specificity and genetic mosaicism of CRISPR/Cas9-mediated gene editing of hemoglobin beta gene in rhesus monkey embryos.
Human molecular genetics - 15 Jul 2017
Midic Uros, Hung Pei-Hsuan, Vincent Kailey A, Goheen Benjamin, Schupp Patrick G, Chen Diane D, Bauer Daniel E, VandeVoort Catherine A, Latham Keith E
Abstract excerpt
Gene editing technologies offer new options for developing novel biomedical research models and for gene and stem cell based therapies. However, applications in many species demand high efficiencies, specificity, and a thorough understanding of likely editing outcomes. To date, overall efficiencies, rates of off-targeting and degree of genetic mosaicism have not been well-characterized for most species, limiting...
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