Developing CRISPR gene editing for treatment of genetic disease: next-generation sequencing to detect and quantify editing at on- and off-target sites

Discovery of the CRISPR technology, originally identified in bacteria, has made it possible to edit our genes. This technology has enormous potential as a new approach for treating inherited diseases in humans. For development of CRISPR therapies that are clinically relevant, we will study new strategies for delivering gene repair kits to cells. For this type of research, it is pivotal to analyze the gene correction activity at a specific, targeted position in the genome. With this application, we aim at providing the infrastructure for performing targeted gene sequencing by next-generation sequencing. This technique provides high data resolution, deep coverage, and detection of low-frequency gene variants.