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Abstract
Improvements in the understanding of human genetics and disease have led to an increased interest in therapeutic genome editing using engineered nucleases. Various approaches have been taken in the past to develop an effective and safe system for sequence specific editing. Compared to earlier nucleases such as ZFN and TALEN, the low cost and ease of producing CRISPR/Cas9 systems has improved the feasibility of therapeutic genome editing. CRISPR/Cas9 genome editing has shown great potential to correct genetic mutations implicated in monogenic diseases and to eradicate viral infections in preclinical studies. Several CRISPR/Cas9-based therapeutics have reached clinical testing, including treatments for inherited red blood cell disorders and Leber Congenital Amaurosis 10, as well as edited T cell-based cancer therapies. Further advances in therapeutic genome editing will require safer and more efficient in vivo CRISPR/Cas9 delivery systems and optimization of HDR efficiency.