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Research papers on CRISPR gene editing

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  1. 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 respiratory

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  3. Therapeutic applications of CRISPR-Cas9 gene editing

    Aditya Bharti, Joann Mudge · 2025 · Frontiers in Genome Editing · 10 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. This review highlights

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  6. 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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  8. 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 dystroph

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  9. 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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