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~ Genome Editing From Modeling Disease to Novel Therapeutics ~

The ability of human pluripotent stem cells (hPSCs) to differentiate into the many specialized cell types of the human body holds great promise for the study and treatment of human disease. Genome editing of hPSCs can allow for more accurate modeling of human diseases by introducing disease-specific mutations, or correct genetic aberrations in patient-derived induced pluripotent stem cell lines. Advances in tools such as transcription activator-like effector nucleases (TALENs) or clustered regularly interspaced short palindromic repeat (CRISPR) technologies have improved the ease and efficiency of genome editing in hPSCs.

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