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Research papers on CRISPR gene editing

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  1. Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

    Ophir Shalem, Neville E. Sanjana, Ella Hartenian, et al. · 2013 · Science · 5,603 citations

    The simplicity of programming the CRISPR (clustered regularly interspaced short palindromic repeats)-associated nuclease Cas9 to modify specific genomic loci suggests a new way to interrogate gene function on a genome-wide scale. We show that lentiviral delivery of a genome-scale CRISPR-Cas9 knockout (GeCKO) library targeting 18,080 genes with 64,751 unique guide sequences enables both negative and positive selection screening in human cells. First, we used the GeCKO library to identify genes essential for cell viability in cancer and pluripotent stem cells. Next, in a melanoma model, we screened for genes whose loss is involved in resistance to vemurafenib, a therapeutic RAF inhibitor. Our

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  4. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

    Julian D. Gillmore, Ed Gane, Jörg Täubel, et al. · 2021 · New England Journal of Medicine · 1,728 citations

    BACKGROUND: . METHODS: After conducting preclinical in vitro and in vivo studies, we evaluated the safety and pharmacodynamic effects of single escalating doses of NTLA-2001 in six patients with hereditary ATTR amyloidosis with polyneuropathy, three in each of the two initial dose groups (0.1 mg per kilogram and 0.3 mg per kilogram), within an ongoing phase 1 clinical study. RESULTS: after a single dose. Serial assessments of safety during the first 28 days after infusion in patients revealed few adverse events, and those that did occur were mild in grade. Dose-dependent pharmacodynamic effects were observed. At day 28, the mean reduction from baseline in serum TTR protein concentration was

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  5. The CRISPR tool kit for genome editing and beyond

    Mazhar Adli · 2018 · Nature Communications · 1,686 citations

    CRISPR is becoming an indispensable tool in biological research. Once known as the bacterial immune system against invading viruses, the programmable capacity of the Cas9 enzyme is now revolutionizing diverse fields of medical research, biotechnology, and agriculture. CRISPR-Cas9 is no longer just a gene-editing tool; the application areas of catalytically impaired inactive Cas9, including gene regulation, epigenetic editing, chromatin engineering, and imaging, now exceed the gene-editing functionality of WT Cas9. Here, we will present a brief history of gene-editing tools and describe the wide range of CRISPR-based genome-targeting tools. We will conclude with future directions and the broa

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  6. CRISPR/Cas9 in Genome Editing and Beyond

    Haifeng Wang, Marie La Russa, Lei S. Qi · 2016 · Annual Review of Biochemistry · 1,213 citations

    The Cas9 protein (CRISPR-associated protein 9), derived from type II CRISPR (clustered regularly interspaced short palindromic repeats) bacterial immune systems, is emerging as a powerful tool for engineering the genome in diverse organisms. As an RNA-guided DNA endonuclease, Cas9 can be easily programmed to target new sites by altering its guide RNA sequence, and its development as a tool has made sequence-specific gene editing several magnitudes easier. The nuclease-deactivated form of Cas9 further provides a versatile RNA-guided DNA-targeting platform for regulating and imaging the genome, as well as for rewriting the epigenetic status, all in a sequence-specific manner. With all of these

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  8. A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

    Jonathan D. Finn, Amy Smith, Mihir Patel, et al. · 2018 · Cell Reports · 876 citations

    The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled significant editing of the mouse transthyretin (Ttr) gene in the liver, with a >97% reduction in serum protein levels that persisted for at least 12 months. These results were achieved with an LNP delivery system that was biodegradable and well tolerated. The LNP delivery system was combined with a sgRNA having a chemical modification pattern that was important for high levels of in vivo activity. The

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  9. CRISPR/Cas9 for genome editing: progress, implications and challenges

    Feng Zhang, Y. Wen, Xiong Guo · 2014 · Human Molecular Genetics · 684 citations

    Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) protein 9 system provides a robust and multiplexable genome editing tool, enabling researchers to precisely manipulate specific genomic elements, and facilitating the elucidation of target gene function in biology and diseases. CRISPR/Cas9 comprises of a nonspecific Cas9 nuclease and a set of programmable sequence-specific CRISPR RNA (crRNA), which can guide Cas9 to cleave DNA and generate double-strand breaks at target sites. Subsequent cellular DNA repair process leads to desired insertions, deletions or substitutions at target sites. The specificity of CRISPR/Cas9-mediated DNA cleavage requires targ

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  11. CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future

    Fathema Uddin, Charles M. Rudin, Triparna Sen · 2020 · Frontiers in Oncology · 582 citations

    A series of recent discoveries harnessing the adaptive immune system of prokaryotes to perform targeted genome editing is having a transformative influence across the biological sciences. The discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) proteins has expanded the applications of genetic research in thousands of laboratories across the globe and is redefining our approach to gene therapy. Traditional gene therapy has raised some concerns, as its reliance on viral vector delivery of therapeutic transgenes can cause both insertional oncogenesis and immunogenic toxicity. While viral vectors remain a key delivery vehicle, CRISPR techno

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  13. CRISPR/Cas9 Genome Editing Tool: A Promising Tool for Therapeutic Applications on Respiratory Diseases

    Sadiya Bi Shaikh, Yashodhar Prabhakar Bhandary · 2020 · Current Gene Therapy · 14 citations

    Respiratory diseases are one of the prime topics of concern in the current era due to improper diagnostics tools. Gene-editing therapy, like Clustered regularly interspaced palindromic repeats- associated nuclease 9 (CRISPR/Cas9), is gaining popularity in pulmonary research, opening up doors to invaluable insights on underlying mechanisms. CRISPR/Cas9 can be considered as a potential gene-editing tool with a scientific community that is helping in the advancement of knowledge in respiratory health and therapy. As an appealing therapeutic tool, we hereby explore the advanced research on the application of CRISPR/Cas9 tools in chronic respiratory diseases such as lung cancer, Acute respirato

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  16. Therapeutic applications of CRISPR-Cas9 gene editing

    Aditya Bharti, Joann Mudge · 2025 · Frontiers in Genome Editing · 6 citations

    CRISPR-Cas9 is a gene editing tool used extensively in biological research that is now making its way into clinical therapies. With the first CRISPR therapy obtaining approval by the United States’ Food and Drug Administration (FDA) in late 2023, we look at clinical trials of emerging therapies involving CRISPR-Cas9, currently the most prevalent CRISPR-based tool in these trials. A CRISPR-based therapy is currently approved for treatment of both sickle-cell anemia and transfusion-dependent β-thalassemia but clinical trials for CRISPR-based therapeutics include a much broader range of targets. CRISPR-Cas9 is being explored to treat cancer, infectious disease, and more. Th

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  17. Recent Advancements in CRISPR-Cas9 Technology for Precision Gene Editing and Therapeutic Applications

    D. Jayarajan, Amit Kumar Dutta, T.Deborah Paripuranam, et al. · 2025 · Journal of Neonatal Surgery · 3 citations

    The CRISPR-Cas9 system has emerged as a transformative tool in genetic engineering, enabling scientists to edit genomes with unprecedented precision, efficiency, and simplicity. Since its adaptation from a prokaryotic immune mechanism to a gene-editing platform, CRISPR-Cas9 has rapidly progressed from bench research to translational and clinical applications. This paper presents a comprehensive review of the most recent advancements in CRISPR-Cas9 technology, particularly focusing on innovations that enhance the accuracy, versatility, and therapeutic utility of gene editing. Key breakthroughs include the development of base editing and prime editing technologies, which allow for single-nucle

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  18. Novel gene-editing technologies: applications of CRISPR-Cas9, base editing, and prime editing in SCID gene therapy

    Greg Crawford, Pervinder Sagoo, H. Bobby Gaspar · 2026 · Journal of Translational Genetics and Genomics · 1 citations

    The use of autologous haematopoietic stem cell gene therapy is increasingly recognised as a promising treatment option for severe combined immunodeficiency diseases (SCID). This approach seeks to correct the underlying genetic cause of SCID conditions, potentially allowing a single treatment to restore a healthy immune system for the lifespan of the patient. To date, such gene therapy has relied on the use of gamma-retroviruses or lentiviruses to deliver genetic material to a patient’s haematopoietic stem cells before reinfusion. This approach has had notable successes in the clinic for conditions including X-linked severe combined immunodeficiency (SCID-X1), Artemis-SCID, and adenosine deam

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  20. ADVANCEMENTS IN CRISPR-CAS9 TECHNOLOGY: IMPLICATIONS FOR GENE EDITING AND THERAPEUTIC APPLICATIONS

    Ezza Fatima, Zia Ur Rehman · 2023 · Biology and Biotechnology Communications

    Gene editing brought about by the CRISPR-Cas9 technology has transformed such an area by introducing the ability to alter genomes by relatively accurate, fast, and affordable methods.  The aims of the review are to cover all the new developments in CRISPR-based systems, including the emergence of Cas12, Cas13, base editing and prime editing technologies. Each of them has improved the target specificity tremendously and the off-target effects considerably too.  We investigated editing efficiency scores of various Cas variants (Cas9, Cas12 and Cas13), and examined over 20 clinical trials that occurred within (2020-2024) and addressed genetic diseases such as sickle cell anemia, muscular dystro

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  21. CRISPR-Cas-Based Epigenome Editing as a Novel Therapeutic Strategy for Metabolic Disorders: Targeting Gene Expression Patterns in Specific Tissues to Treat Diabetes and Obesity

    Ashfaq Ahmad · 2023 · Diabetes & Obesity International Journal

    Metabolic disorders such as diabetes and obesity are major public health concerns that affect millions of people worldwide. Epigenetic modifications have been implicated in the development and progression of these disorders, making them attractive targets for therapeutic intervention. The emergence of Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPRCas-based) epigenome editing technology has revolutionized the field of gene editing and holds great promise for the treatment of metabolic disorders. However, there are still significant challenges and ethical concerns that must be addressed before this technology can be safely and effectively used in clinical applications. This

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