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Papers on “CRISPR gene editing therapeutic applications”

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  1. Appropriate Delivery of the CRISPR/Cas9 System through the Nonlysosomal Route: Application for Therapeutic Gene Editing

    Hang Yin, Xiaoling Yuan, Lihua Luo, et al. · 2020 · Advanced Science · 43 cites

    The development of gene delivery has attracted increasing attention, especially when the introduction and application of the CRISPR/Cas9 gene editing system appears promising for gene therapy. However, ensuring biosafety and high gene editing efficiency at the same time poses a great challenge for its in vivo applications. Herein, a pardaxin peptide (PAR)-modified cationic liposome (PAR-Lipo) is developed. The results are indicative that significantly enhanced gene editing efficiency can be obtained through the mediation of PAR-Lipos compared to non-Lipos (non-PAR-modified liposomes) and Lipofectamine 2000, owing to its protection toward carried nucleotide by the prevention of lysosomal capt

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  2. Therapeutic applications of CRISPR /Cas9 gene editing technology for the treatment of ocular diseases

    Yogapriya Sundaresan, Sam Yacoub, Bindu Kodati, et al. · 2023 · FEBS Journal · 24 cites

    Ocular diseases are a highly heterogeneous group of phenotypes, caused by a spectrum of genetic variants and environmental factors that exhibit diverse clinical symptoms. As a result of its anatomical location, structure and immune privilege, the eye is an ideal system to assess and validate novel genetic therapies. Advances in genome editing have revolutionized the field of biomedical science, enabling researchers to understand the biology behind disease mechanisms and allow the treatment of several health conditions, including ocular pathologies. The advent of clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing facilitates efficient and specific genetic mo

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  3. 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 cites

    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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  4. Therapeutic applications of CRISPR-Cas9 gene editing

    Aditya Bharti, Joann Mudge · 2025 · Frontiers in Genome Editing · 11 cites

    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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