Abstract
Myriads of genetic mutations, including base substitutions, deletions, and insertions as well as chromosome structural variations, have been detected in many human diseases. Although current combination of genomics and bioinformatics has contributed greatly to understanding the genetics of these disorders, it remains challenging to ensure the causal functions of each mutation, and then to further investigate the underlying mechanism and to develop therapeutic strategies. Animal models generated by genome engineering are the key to address these issues. In this review, we will first revisit the limitation of conventional gene editing tools and mouse models generated in the past. We will then introduce a novel tool, base editors (BEs), which present a new promising approach to establish pathogenically relevant animal models. Finally, we will discuss the application of BEs in non-human primates and share our perspectives on future development of base-editing techniques.
Highlights
BASE EDITORS: A NOVEL GENOME ENGINEERING TOOL Genetically engineered mouse models of human diseases have long been used in understanding the underlying mechanisms of the clinically relevant genetic disorders
We will discuss the application of base editors (BEs) in non-human primates and share our perspectives on future development of base-editing techniques
Novel single base editing tools named BEs created by fusing the cytosine/adenine deaminase to the N-terminus of Cas9 nickase have been constructed to convert A to G or C to T at specific sites, without causing DSB and with minimized off-target [1] (Fig. 1)
Summary
BASE EDITORS: A NOVEL GENOME ENGINEERING TOOL Genetically engineered mouse models of human diseases have long been used in understanding the underlying mechanisms of the clinically relevant genetic disorders. We will first revisit the limitation of conventional gene editing tools and mouse models generated in the past. We will introduce a novel tool, base editors (BEs), which present a new promising approach to establish pathogenically relevant animal models.
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