Genome editing technology enables manipulations at genome level where DNA is replaced, deleted or inserted in a living organism. Classic genome editing approaches depend on homology-directed repair and the totipotency of stem cells to facilitate the modification of the individual gene. The Nobel Prize in Physiology or Medicine 2007 was awarded to the discoveries of principles for introducing specific gene modifications in mice by the use of embryonic stem cells. Because the traditional HDR approach has disadvantages of low efficiency, high technical requirements and high cost, which seriously restricted its applications in large-scale genomic manipulation research. In 2013 the discovery of the type II CRISPR-Cas9 system has promoted the development of precise genetic modifications. This new RNA-mediated DNA editing approach opens up new avenues for the application of genome editing technology in Animal model construction, genetic disease treatment and agricultural breeding.
Genome editing technology——Generation of Animal models
CRISPR-Cas9 system performs precise targeting and editing a specific DNA sequence of interest via a programmable mechanism, and provides a versatile approach to establish transgenic animal models. While mouse models have been widely used, the CRISPR-Cas9 gene-editing approach has been established in many other animal models, including worm, rat, rabbit, pig and monkey. New mouse models can be generated with CRISPR-Cas9 by injecting Cas9 mRNA and guide RNAs (sgRNA) directly into mouse embryos to generate precise genomic edits into specific loci with an efficiency of 100%. CRISPR-Cas9 mediated targeting and editing has facilitated the generation of knockin and knockout mouse models, dramatically decreases the time and resource consumption comparing to traditional methods.
Genome editing technology——Curing genetic diseases
Although CRISPR-Cas9 has already has been widely used as a research tool, a particularly exciting future direction is the development of CRISPR-Cas9 as a therapeutic technology for treating genetic disorders. Researchers at Chinese Academy of Sciences reported successful correction of disease-causing mutations in cataract mouse models via the CRISPR-Cas9 system. Upon injection of CRISPR-Cas9 into zygotes, 1/3 genetic defect in the cataract mouse model could be corrected at the organism level and more importantly the corrected trait was successfully transmitted to the next generation through the germline.
Genome editing technology——Agricultural breeding
The CRISPR-Cas9 technology opens up exciting possibilities for creating crop varieties with desirable traits without introducing foreign DNA. Precision breeding crops with desirable traits, such as disease resistance and drought tolerance not only help reduce pesticide, fertilizer and water usage, but also improve food quality and safety. Researchers at Penn State University created mushrooms with reduced production of a specific enzyme that causes mushrooms to blemishes caused by handling or mechanical harvesting. It becomes the first CRISPR-edited organism to receive a green light from the US government, means that the mushroom can be cultivated and sold without passing through the agency’s regulatory process.
CRISPR-Cas9 is an emerging technology that enables precise genome modification without introducing foreign genes. This transformative tool holds great promises to revolutionize biological research and expand our ability correcting the genetic causes behind many diseases.
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