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Research papers on CRISPR gene editing

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  1. Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications

    Chang Liu, Li Zhang, Hao Liu, et al. · 2017 · Journal of Controlled Release · 607 citations

    ABSTRACT The CRISPR‐Cas9 genome‐editing system is a part of the adaptive immune system in archaea and bacteria to defend against invasive nucleic acids from phages and plasmids. The single guide RNA (sgRNA) of the system recognizes its target sequence in the genome, and the Cas9 nuclease of the system acts as a pair of scissors to cleave the double strands of DNA. Since its discovery, CRISPR‐Cas9 has become the most robust platform for genome engineering in eukaryotic cells. Recently, the CRISPR‐Cas9 system has triggered enormous interest in therapeutic applications. CRISPR‐Cas9 can be applied to correct disease‐causing gene mutations or engineer T cells for cancer immunotherapy. The first c

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  3. Current approaches in CRISPR-Cas9 mediated gene editing for biomedical and therapeutic applications

    Gargi Bhattacharjee, Nisarg Gohil, Khushal Khambhati, et al. · 2022 · Journal of Controlled Release · 78 citations

    A single gene mutation can cause a number of human diseases that affect quality of life. Until the development of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas) systems, it was challenging to correct a gene mutation to avoid disease by reverting phenotypes. The advent of CRISPR technology has changed the field of gene editing, given its simplicity and intrinsic programmability, surpassing the limitations of both zinc-finger nuclease and transcription activator-like effector nuclease and becoming the method of choice for therapeutic gene editing by overcoming the bottlenecks of conventional gene-editing techniques. Currently, there is no

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  4. Recent advances in the delivery and applications of nonviral CRISPR/Cas9 gene editing

    Frazer H. Sinclair, Anjuman A. Begum, Charles C. Dai, et al. · 2023 · Drug Delivery and Translational Research · 74 citations

    The CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 genome editing system has been a major technological breakthrough that has brought revolutionary changes to genome editing for therapeutic and diagnostic purposes and precision medicine. With the advent of the CRISPR/Cas9 system, one of the critical limiting factors has been the safe and efficient delivery of this system to cells or tissues of interest. Several approaches have been investigated to find delivery systems that can attain tissue-targeted delivery, lowering the chances of off-target editing. While viral vectors have shown promise for in vitro, in vivo and ex vivo delivery of CRISPR/Cas9, their further cli

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  5. Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

    Fan Wu, Nei Li, Yudian Xiao, et al. · 2025 · Small Methods · 57 citations

    Gene editing has emerged as a promising therapeutic option for treating genetic diseases. However, a central challenge in the field is the safe and efficient delivery of these large editing tools, especially in vivo. Lipid nanoparticles (LNPs) are attractive nonviral vectors due to their low immunogenicity and high delivery efficiency. To maximize editing efficiency, LNPs should efficiently protect gene editing components against multiple biological barriers and release them into the cytoplasm of target cells. In this review, the widely used CRISPR gene editing systems are first overviewed. Then, each component of LNPs, as well as their effects on delivery, are systematically discussed. Foll

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  6. CRISPR Gene Editing of Human Primary NK and T Cells for Cancer Immunotherapy

    Ezgi Elmas, Noushin Saljoughian, Marcelo de Souza Fernandes Pereira, et al. · 2022 · Frontiers in Oncology · 44 citations

    Antitumor activity of immune cells such as T cells and NK cells has made them auspicious therapeutic regimens for adaptive cancer immunotherapy. Enhancing their cytotoxic effects against malignancies and overcoming their suppression in tumor microenvironment (TME) may improve their efficacy to treat cancers. Clustered, regularly interspaced short palindromic repeats (CRISPR) genome editing has become one of the most popular tools to enhance immune cell antitumor activity. In this review we highlight applications and practicability of CRISPR/Cas9 gene editing and engineering strategies for cancer immunotherapy. In addition, we have reviewed several approaches to study CRISPR off-target effect

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  7. 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 citations

    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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  8. Transforming Pharmacogenomics and CRISPR Gene Editing with the Power of Artificial Intelligence for Precision Medicine

    A. Srivastav, M. Mishra, James W. Lillard, et al. · 2025 · Pharmaceutics · 31 citations

    Background: Advancements in pharmacogenomics, artificial intelligence (AI), and CRISPR gene-editing technology are revolutionizing precision medicine by enabling highly individualized therapeutic strategies. Artificial intelligence-driven computational techniques improve biomarker discovery and drug optimization while pharmacogenomics helps to identify genetic polymorphisms affecting medicine metabolism, efficacy, and toxicity. Genetically editing based on CRISPR presents a precise method for changing gene expression and repairing damaging mutations. This review explores the convergence of these three fields to enhance improved precision medicine. Method: A methodical study of the current li

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  9. 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 citations

    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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  10. Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR‐Cas12f1 System Delivered with All‐in‐One Adeno‐Associated Virus

    T. Cui, Bingyu Cai, Yao Tian, et al. · 2024 · Advanced Science · 18 citations

    CRISPR‐based gene therapies are making remarkable strides toward the clinic. But the large size of most widely used Cas endonucleases including Cas9 and Cas12a restricts their efficient delivery by the adeno‐associated virus (AAV) for in vivo gene editing. Being exceptionally small, the recently engineered type V‐F CRISPR‐Cas12f1 systems can overcome the cargo packaging bottleneck and present as strong candidates for therapeutic applications. In this study, the pairwise editing efficiencies of different engineered Cas12f1/sgRNA scaffold combinations are systemically screened and optimized, and the CasMINI_v3.1/ge4.1 system is identified as being able to significantly boost the gene editing a

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  11. An extensive review to facilitate understanding of CRISPR technology as a gene editing possibility for enhanced therapeutic applications

    Indra Rautela, Pooja Uniyal, Priya Thapliyal, et al. · 2021 · Gene · 14 citations

    CRISPR are the sequences in bacterial and archaeal genome which provide resistance against viral infections. They might be the natural part of bacterial genomes for providing protection against viruses like bacteriophages but science has successfully achieved their use in the benefit of man-kind by using them for the treatment of deadly diseases like cancer, AIDS or genetic disorders like sickle cell disease and Leber congenital amaurosis. CRISPR system is majorly divided into two classes i.e class I and class II, of which the class II CRISPR/Cas9 system performs site specific cleavage of DNA with a guide RNA Cas12 (Cpf1). With the new emerging discoveries it is being found that CRISPR not o

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  12. 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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  13. Advances in Therapeutic Applications of CRISPR Genome Editing for Spinal Pain Management

    Chan-Young Kang, K. W. Been, Myoung-Hee Kang, et al. · 2025 · Neurospine · 12 citations

    Neuropathic pain remains a significant clinical challenge due to the limited efficacy and sustainability of existing pharmacological treatments, underscoring the urgent need for mechanism-based therapeutic strategies. In recent years, gene-targeted interventions have emerged as promising modalities capable of modulating key molecular pathways implicated in chronic pain. Approaches such as antisense oligonucleotides and RNA interference have demonstrated encouraging preclinical results by selectively downregulating pain-associated genes. Based on these developments, genome-editing technologies—particularly the clustered regularly interspaced short palindromic repeats (CRISPR) system—have enab

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  14. Therapeutic applications of CRISPR-Cas9 gene editing

    Aditya Bharti, Joann Mudge · 2025 · Frontiers in Genome Editing · 11 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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  15. The Future of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas9 Gene Therapy in Cardiomyopathies: A Review of Its Therapeutic Potential and Emerging Applications

    Ali Moradi, Sina Khoshniyat, Tochukwu Nzeako, et al. · 2025 · Cureus · 10 citations

    Cardiomyopathies, among the leading causes of heart failure and sudden cardiac death, are often driven by genetic mutations affecting the heart's structural proteins. Despite significant advancements in understanding the genetic basis of hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM), and arrhythmogenic right ventricular cardiomyopathy (ARVC), effective long-term therapies remain limited. The advent of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (Cas9) gene editing offers a promising therapeutic strategy to address these genetic disorders at their root. CRISPR-Cas9 enables precise modification of pathogenic variants (PVs

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  16. Therapeutic applications of CRISPR/Cas9 mediated targeted gene editing in acute lymphoblastic leukemia: current perspectives, future challenges, and clinical implications

    Alan Jhones Barbosa Assis, Brunna Letícia de Oliveira Santana, A. Gualberto, et al. · 2023 · Frontiers in Pharmacology · 9 citations

    Acute Lymphoblastic Leukemia (ALL) is the predominant hematological malignancy in pediatric populations, originating from B- or T-cell precursors within the bone marrow. The disease exhibits a high degree of heterogeneity, both at the molecular level and in terms of clinical presentation. A complex interplay between inherited and acquired genetic alterations contributes to disease pathogenesis, often resulting in the disruption of cellular functions integral to the leukemogenic process. The advent of CRISPR/Cas9 as a gene editing tool has revolutionized biological research, underscoring its potential to modify specific genomic loci implicated in cancer. Enhanced understanding of molecular al

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  18. Viral Delivery of Compact CRISPR-Cas12f for Gene Editing Applications

    A. Sharrar, Zuriah Meacham, Johanna Staples-Ager, et al. · 2024 · The CRISPR Journal · 8 citations

    Treating human genetic conditions in vivo requires efficient delivery of the CRISPR gene editing machinery to the affected cells and organs. The gene editing field has seen clinical advances with ex vivo therapies and with in vivo delivery to the liver using lipid nanoparticle technology. Adeno-associated virus (AAV) serotypes have been discovered and engineered to deliver genetic material to nearly every organ in the body. However, the large size of most CRISPR-Cas systems limits packaging into the viral genome and reduces drug development flexibility and manufacturing efficiency. Here, we demonstrate efficient CRISPR gene editing using a miniature CRISPR-Cas12f system with expanded genome

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  20. Refined control of CRISPR-Cas9 gene editing in Clostridium sporogenes: the creation of recombinant strains for therapeutic applications

    A. Kubiak, Luuk Claessen, Yanchao Zhang, et al. · 2023 · Frontiers in Immunology · 7 citations

    Despite considerable clinical success, the potential of cancer immunotherapy is restricted by a lack of tumour-targeting strategies. Treatment requires systemic delivery of cytokines or antibodies at high levels to achieve clinically effective doses at malignant sites. This is exacerbated by poor penetration of tumour tissue by therapeutic antibodies. High-grade immune-related adverse events (irAEs) occur in a significant number of patients (5-15%, cancer- and therapeutic-dependent) that can lead to lifelong issues and can exclude from treatment patients with pre-existing autoimmune diseases. Tumour-homing bacteria, genetically engineered to produce therapeutics, is one of the approaches tha

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  21. Advances in CRISPR Base Editing: From Molecular Evolution to Therapeutic Applications in Genomic Medicine

    Melike Aliciaslan, Ezgi Erbasan, Fulya Erendor, et al. · 2026 · Journal of Cellular and Molecular Medicine · 5 citations

    CRISPR‐Cas9 systems revolutionized gene editing, but inherent drawbacks, namely DNA double‐strand breaks (DSBs) and the difficulty of achieving precise repairs (due to low HDR efficiency), led researchers to invent new, more accurate gene editing tools. Base editing represents a significant leap forward, enabling targeted single‐nucleotide conversions directly on the DNA without DSBs or donor templates. The core technology involves fusing catalytically dead or nickase Cas proteins to DNA deaminase enzymes. Cytosine base editors (CBEs) convert C•G to T•A pairs, while adenine base editors (ABEs) change A•T to G•C. These editors exploit the deaminase function within the R‐loop structure formed

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

    D. Jayarajan, Amit Kumar Dutta, Dr T. Deborah Paripuranam, et al. · 2025 · Journal of Neonatal Surgery · 4 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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  23. Metal-Organic Frameworks for CRISPR/Cas9 Gene Editing Delivery: Innovations in Therapeutic and Diagnostic Applications.

    Zheng-Xian Su, Zeyang Liang, Qiong Wu, et al. · 2025 · Acta biomaterialia · 4 citations

    CRISPR/Cas gene editing technology demonstrates significant promise in the treatment of various diseases, and a precise, efficient and safe delivery system is a key to realize gene therapy. Although traditional viral vectors can achieve superior transfection efficiency, viruses suffer from low reproduction efficiency and the risk of random gene integration, further limiting their wide application. Notably, metal-organic frameworks (MOFs), with tunable pore structure, easy surface chemical modification, good biocompatibility and physiological stability, have drawn much attention in the domain of targeted delivery of gene editing systems. Compared to lipid nanoparticles (LNPs) and extracellula

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