Abstract
The development of novel genome editing tools has unlocked new opportunities that were not previously possible in basic and biomedical research. During the last two decades, several new genome editing methods have been developed that can be customized to modify specific regions of the genome. However, in the past couple of years, many newer and more exciting genome editing techniques have been developed that are more efficient, precise, and easier to use. These genome editing tools have helped to improve our understanding of genetic disorders by modeling them in cells and animal models, in addition to correcting the disease-causing mutations. Among the genome editing tools, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system has proven to be the most popular one due to its versatility and has been successfully used in a wide variety of laboratory animal models and plants. In this review, we summarize the customizable nucleases currently used for genome editing and their uses beyond the modification of genome. We also discuss the potential future applications of gene editing tools for both basic research and clinical purposes.
Highlights
The ability to precisely manipulate the genome revolutionized molecular biology, and opened several areas of biotechnology that are useful for research, agriculture, and medicine
This review aims to provide an overview of the recent developments in and applications of engineered nucleases that have helped lay the groundwork for their use for genome editing in various animal models, and to correct the genetic mutations in human cells for clinical use
We developed an non-homology end joining (NHEJ)-based homology independent strategy using clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated 9 (Cas9), named homology-independent target integration (HITI), for the integration of transgene in non-dividing cells [29,37]
Summary
The ability to precisely manipulate the genome revolutionized molecular biology, and opened several areas of biotechnology that are useful for research, agriculture, and medicine. Initial gene targeting primarily depended on homologous recombination to insert exogenous DNA sequences in the human cells [1]. Vectors with homology arms proved efficient in modifying the chromosomal target sequences [5] The mechanism behind this observation is not entirely clear. During the repair of DSBs, the donor template with homology arms can get inserted at the site of repair using homology-directed repair (HDR) and dramatically increases the gene targeting efficiency [6]. The discovery of customizable nucleases that can be programmed to induce DSBs at desired loci on the genome dramatically increased the efficiency of homologous recombination, leading to another revolution in gene editing with much broader implications in several different fields. We review the use of genome editing tools in human embryos
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