New gene editing technology
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Advances in Gene Editing Technology
Introduction to Gene Editing Technologies
Gene editing technologies have revolutionized the field of genetic research and therapy by enabling precise modifications to the human genome. These technologies allow for the correction of mutations, addition of therapeutic genes, and removal of deleterious sequences, offering new therapeutic possibilities1. The primary tools used in gene editing include zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), meganucleases, and the CRISPR/Cas9 system1 2.
Mechanisms and Platforms of Gene Editing
Nuclease-Based Platforms
The main platforms for gene editing are based on engineered nucleases that create targeted double-stranded breaks in DNA. These include:
- Zinc Finger Nucleases (ZFNs): These are engineered proteins that create double-strand breaks at specific locations in the genome, which can then be repaired by the cell's natural repair mechanisms1 4.
- Transcription Activator-Like Effector Nucleases (TALENs): Similar to ZFNs, TALENs are customizable proteins that can target and cut specific DNA sequences1 4.
- CRISPR/Cas9 System: This system uses RNA-guided nucleases to create precise cuts in the DNA, allowing for targeted modifications. It has become the most widely used tool due to its simplicity and efficiency1 2 4.
DNA Repair Mechanisms
Once a double-strand break is introduced, the cell employs various repair mechanisms to fix the break, which can be harnessed for gene editing:
- Non-Homologous End Joining (NHEJ): This is a quick repair process that often results in insertions or deletions (indels) at the break site2 4.
- Homology-Directed Repair (HDR): This mechanism uses a homologous sequence as a template for repair, allowing for precise modifications2 4.
- Microhomology-Mediated End Joining (MMEJ): This is another repair pathway that can be used for precise editing2 4.
Advances in Delivery Technologies
Efficient delivery of gene-editing components is crucial for their success. Both viral and non-viral delivery methods are being developed and optimized:
- Viral Delivery: Adeno-associated viruses (AAV) are commonly used for delivering gene-editing tools due to their high efficiency3.
- Non-Viral Delivery: Lipid nanoparticles and cell-penetrating peptides are being explored as safer alternatives to viral vectors3.
Clinical Applications and Challenges
Therapeutic Applications
Gene editing has shown promise in various therapeutic areas:
- Antiviral Strategies: Gene editing is being explored for its potential to target and eliminate viral genomes, such as HIV1 2.
- Immunotherapies: Modifying immune cells to better target cancer cells is a significant area of research1 2.
- Monogenic Disorders: Diseases caused by single-gene mutations, such as sickle cell disease and β-thalassemia, are prime candidates for gene editing therapies2 6.
Challenges and Future Prospects
Despite the progress, several challenges remain:
- Off-Target Effects: Unintended modifications to the genome can occur, but advances in high-fidelity Cas9 variants are helping to minimize these effects2.
- Delivery Efficiency: Ensuring that gene-editing tools reach the target cells efficiently and safely is an ongoing challenge3.
- Ethical and Regulatory Issues: The potential for germline editing raises significant ethical concerns that need to be addressed1.
Conclusion
Gene editing technologies have made significant strides, offering new possibilities for treating genetic disorders and advancing biomedical research. While challenges remain, ongoing research and technological advancements continue to improve the precision, efficiency, and safety of these powerful tools. The future of gene editing holds great promise for transforming medicine and improving human health.
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Most relevant research papers on this topic
Genome-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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Gene-editing technology has advanced, enabling precise gene modifications for cancer treatment, HIV therapy, and genetic disorders, but challenges remain in clinical implementation.
Highly Cited2019·81citations·M. Broeders et al.·iScience
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Delivery 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 Cited2017·447citations·Hao Yin et al.·Nature Reviews Drug Discovery
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Precision gene editing technology and applications in nephrology
Precision gene editing technology has the potential to accelerate kidney research and advance clinical practice, despite challenges in anatomical barriers and immune responses.
2018·43citations·Zachary WareJoncas et al.·Nature Reviews Nephrology
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Gene editing for cell engineering: trends and applications
Modern gene editing tools like Crispr-Cas9 enable efficient and cost-effective genetic manipulation of yeast and mammalian cells, leading to faster and more consistent product development and affordability for recombinant therapeutic proteins.
Highly Cited2017·99citations·Sanjeev K Gupta et al.·Critical Reviews in Biotechnology
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Applications 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.
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New advances in CRISPR/Cas-mediated precise gene-editing techniques
Recent advances in CRISPR/Cas-mediated precise gene-editing techniques improve efficiency, enabling the generation of models of human genetic diseases.
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CRISPR-Cas systems for editing, regulating and targeting genomes
CRISPR-Cas9 technology revolutionizes genome editing, enabling rapid, efficient gene modification in various cell types and organisms, potentially revolutionizing biological research and advancing molecular therapeutics for human diseases.
Rigorous JournalHighly Cited2014·2942citations·Jeffry D. Sander et al.·Nature Biotechnology
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Multiplexed CRISPR technologies for gene editing and transcriptional regulation
Multiplexed CRISPR technologies enhance genetic editing and transcriptional regulation, benefiting applications like cellular recorders, genetic circuits, biosensors, and large-scale genome engineering.
Highly Cited2020·304citations·Nicholas S. McCarty et al.·Nature Communications
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Updates on gene editing and its applications.
Gene editing advances offer potential for improving crops and de novo domesticating new crops, with potential applications in basic and applied plant biology research.
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