Genome editing
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Advances and Applications of Genome Editing
Introduction to Genome Editing Technology
Genome editing is a transformative technology that allows for the precise manipulation of DNA sequences within living organisms. This technology has evolved significantly, from the use of chemical and physical mutagenic agents to advanced biological tools such as designed sequence-specific nucleases (SSNs) and Oligonucleotide Directed Mutagenesis (ODM)1. The development of programmable nucleases, including zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR/Cas9, has revolutionized the field, enabling targeted modifications with high precision3 8.
Genome Editing in Plants
Genome editing has been extensively applied in plant biotechnology to enhance crop traits such as yield, pest resistance, and nutrient content. Tools like CRISPR/Cas9 have been used to create knock-out (KO) or knock-in (KI) edits, leading to the development of crops like Cibus' SU Canola™, which is considered non-genetically modified by the USDA1. These advancements hold significant potential for crop improvement, allowing for the precise characterization of gene functions and the enhancement of agricultural traits7.
Therapeutic Applications of Genome Editing
CRISPR Technology in Human Therapy
CRISPR technology has shown immense promise in therapeutic applications, offering the potential to cure genetic diseases by precisely altering DNA sequences. This technology is already in clinical trials for several diseases, highlighting both its opportunities and challenges2. The ability to create site-specific DNA double-strand breaks (DSBs) and leverage the cell's endogenous repair mechanisms has made genome editing a powerful tool for treating a wide variety of diseases6.
Advances in Gene and Cell Therapy
Genome editing has redefined gene therapy by enabling the precise correction of mutations, addition of therapeutic genes, and removal of deleterious sequences. This approach has been applied to antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders10. The progress in this field underscores the potential of genome editing to transform gene and cell therapy, although challenges such as delivery efficiency and safety remain4 5.
Delivery Technologies for Genome Editing
Efficient and safe delivery of genome-editing tools into target cells is crucial for their success. Both viral and non-viral delivery methods are being explored to enhance the uptake and functionality of these tools in cells. Advances in delivery technologies are critical for the clinical application of genome editing, ensuring that the tools reach their target cells and perform their intended functions effectively4.
Ethical and Regulatory Considerations
The rapid advancement of genome editing technologies has raised important ethical and regulatory questions. The potential to edit the human germline, for instance, poses significant moral and societal challenges. Ensuring responsible use of these technologies is paramount to prevent misuse and address ethical concerns2 6. Additionally, regulatory frameworks, particularly in regions like the EU, will play a crucial role in determining the extent to which genome editing can be applied in agriculture and medicine9.
Conclusion
Genome editing represents a groundbreaking advancement in biotechnology, with wide-ranging applications in agriculture and medicine. The development of precise and efficient genome-editing tools like CRISPR/Cas9 has opened new avenues for crop improvement and the treatment of genetic diseases. However, the successful implementation of these technologies will depend on overcoming delivery challenges, addressing ethical concerns, and navigating regulatory landscapes. As research progresses, genome editing holds the promise of significant benefits for both human health and agricultural productivity.
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Most relevant research papers on this topic
Genome Editing of Plants
Genome editing of plants, using tools like designed sequence-specific nucleases, has the potential to revolutionize agriculture by creating non-genetically modified crops like SU CanolaTM.
Highly CitedTHE PROMISE AND CHALLENGE OF THERAPEUTIC GENOME EDITING
CRISPR technology has the potential to treat, cure, and prevent genetic diseases, but responsible use is crucial to ensure its responsible development and application.
Rigorous JournalHighly CitedApplications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
Genome editing technologies, such as ZFNs, TALENs, and CRISPR/Cas9, show potential for targeted therapy and research in various human diseases, with potential for future clinical trials.
Rigorous JournalHighly CitedDelivery technologies for genome editing
Effective and safe delivery technologies are crucial for efficient genome editing, aiding in biomedical research, drug discovery, and gene therapy.
Highly CitedGenome editing at the crossroads of delivery, specificity, and fidelity.
Genome editing progresses with improved delivery of designer nucleases and donor DNA templates, leading to increased specificity and fidelity in gene knockouts and gene knock-ins.
Highly CitedGenome Editing: A New Approach to Human Therapeutics.
Genome editing is a promising strategy for treating human diseases, with potential applications in a wide variety of diseases and a potential for ethical considerations in human germline engineering.
Highly CitedApplications and potential of genome editing in crop improvement
Genome editing in plants offers potential for crop improvement, enhancing adaptation, resilience, and end-use, with potential for commercialization and integration with conventional and new-age crop breeding strategies.
Rigorous JournalHighly CitedGenome editing comes of age
CRISPR-Cas9 genome editing has revolutionized the field by rapidly implementing targeted genetic changes in living cells and organisms.
Highly CitedRegulatory uncertainty over genome editing
EU regulation on genome editing could boost crop yields, protect against pests and diseases, and enhance nutrient content, but its impact on applied plant research and crop breeding depends on how it is regulated.
Highly CitedGenome-editing Technologies for Gene and Cell Therapy
Genome-editing technologies enable precise manipulation of human genomes for therapeutic effects in gene and cell therapy, offering potential for antiviral strategies, immunotherapies, and treating monogenic hereditary disorders.
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