The Development and Application of a Base Editor in Biomedicine.

Fang Wang,Yi Wang,Yuyu Niu,Yuqiang Zeng

doi:10.1155/2020/2907623

Fang Wang, Yi Wang + Show 2 more

Open Access

https://doi.org/10.1155/2020/2907623

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Abstract

Using a base editor to generate monogenic disease models and correct pathogenic point mutations is a breakthrough technology for exploration and treatment of human diseases. As a burgeoning approach for genomic modification, the fused CRISPR/Cas9 with various deaminase separately has significantly increased the efficiency of producing a precise point mutation with minimal insertions or deletions (indels). Along with the flexibility and efficiency, a base editor has been widely used in many fields. This review discusses the recent development of a base editor, including evolution and advance, and highlights the applications and challenges in the field of gene therapy. Depending on rapid improvement and optimization of gene editing technology, the prospect of base editor is immeasurable.

Highlights

Benefit from the progress of gene therapy, we are entering an era in which genome editing tools could be used to manipulate gene sequences flexibly and precisely
Afterwards, some engineered DNA-cleaving enzymes were discovered, including zincfinger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). Both of them demonstrated the potentiality of therapeutic genome editing, they required a lot of time and labor
CRISPR/Cas9 protein-RNA complexes were recruited to target DNA sequence via base pairing with a specified single guide RNA and natively create a double strand breaks (DSBs), triggering cellular DNA repair by nonhomologous end joining (NHEJ) or homology-directed repair (HDR) to achieve genome editing eventually [7, 8]

Summary

Introduction

Benefit from the progress of gene therapy, we are entering an era in which genome editing tools could be used to manipulate gene sequences flexibly and precisely. CRISPR/Cas protein-RNA complexes were recruited to target DNA sequence via base pairing with a specified single guide RNA (sgRNA) and natively create a double strand breaks (DSBs), triggering cellular DNA repair by nonhomologous end joining (NHEJ) or homology-directed repair (HDR) to achieve genome editing eventually [7, 8]. Based on this property of CRISPR/Cas, scientists have developed a variety of derivatives according to different gene editing requirements. We will elaborate the development and application of a base editor in gene therapy

The Evolution of Base Editor

G APOBEC1

The Application of Base Editor in Biomedicine

Findings

Conclusions