Current gene therapy最新文献

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Imaged Capillary Isoelectric Focusing (icIEF) Platform for Characterization of Charge Variants of Adeno-Associated Virus (AAV) Capsids and Impact on Their Transduction Efficiency. 成像毛细管等电聚焦(icIEF)平台表征腺相关病毒(AAV)衣壳的电荷变异及其对其转导效率的影响
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-04-15 DOI: 10.2174/0115665232369708250327081305
Brandon Hoyle, Dhimiter Bello, Jonathan Hill, Soumita Das, Jonghan Kim
{"title":"Imaged Capillary Isoelectric Focusing (icIEF) Platform for Characterization of Charge Variants of Adeno-Associated Virus (AAV) Capsids and Impact on Their Transduction Efficiency.","authors":"Brandon Hoyle, Dhimiter Bello, Jonathan Hill, Soumita Das, Jonghan Kim","doi":"10.2174/0115665232369708250327081305","DOIUrl":"https://doi.org/10.2174/0115665232369708250327081305","url":null,"abstract":"<p><strong>Objective: </strong>Adeno-Associated Virus (AAV) vectors are comprised of a capsid protein encapsulating a Deoxyribonucleic Acid (DNA) transgene that has been used in the gene therapy field showing potential to treat a range of genetic diseases. Methods in the field of gene therapy should be optimized or enhanced to deepen understanding of AAVs, specifically around charge heterogeneity of capsid species.</p><p><strong>Methods: </strong>In this study, a versatile approach was presented for investigating the charge heterogeneity of Adeno-Associated Virus (AAV) capsid proteins of a variety of serotypes. This method employs Imaged Capillary Isoelectric Focusing (icIEF) coupled with native fluorescence imaging detection and has undergone exhaustive validation.</p><p><strong>Results: </strong>Demonstrating its platform nature, this method analyzed seven different AAV serotypes from multiple manufacturing platforms. The distinctive profiles generated for each AAV serotype serve as valuable indicators for both identity confirmation and stability assessment. It was shown that thermal stress and pH conditions play a role in increasing acidic charged variants over time, affecting the charge heterogeneity of AAVs, which can be serotype-specific. Reverse phase LC-MS was used to identify and confirm the increased presence of Post-Translational Modifications (PTMs) that are linked to increasing acidic species variants relative to non-stressed AAVs.</p><p><strong>Conclusion: </strong>These PTMs have biological consequences reflected in the diminished expression of the protein of interest in vitro. This cIEF method successfully analyzed a variety of AAV serotypes, and increasing trends of acidic variants led to reduced in vitro potency.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144062560","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Next-Generation Whole-Exome Pattern: Advanced Methods and Clinical Significance. 下一代全外显子组模式:先进的方法和临床意义。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-04-14 DOI: 10.2174/0115665232356780250331181436
Sumel Ashique, Anas Islam, Navjot Kaur Sandhu, Bhavinee Sharma, Rashmi Pathak, Himanshu Sharma
{"title":"Next-Generation Whole-Exome Pattern: Advanced Methods and Clinical Significance.","authors":"Sumel Ashique, Anas Islam, Navjot Kaur Sandhu, Bhavinee Sharma, Rashmi Pathak, Himanshu Sharma","doi":"10.2174/0115665232356780250331181436","DOIUrl":"https://doi.org/10.2174/0115665232356780250331181436","url":null,"abstract":"<p><p>NGS (Next-generation sequencing) has emerged as the primary approach for gene finding in uncommon hereditary disorders. Targeted gene panels, whole genome sequencing (WGS), and whole exome sequencing (WES) are uses of next-generation sequencing and other related technologies. It is possible to explain personal or individual genome sequencing using NGS technology, as well as to detect disease-causing mutations using NGS findings. NGS, deep sequencing or massively parallel are similar words that describe a method of DNA sequencing leading to revolutionary change in genomic research. Due to its cost-effectiveness, Whole-Exome sequencing (WES) using Next-Generation Sequencing (NGS) is becoming increasingly popular in the field of human genetics. As a diagnostic tool, this technology can reduce the duration of the diagnostic process for several patients and has mostly made a significant contribution to the identification of new genes responsible for causing diseases. Considering the diverse range of phenotypic presentations of the diagnosis, NGS has the potential to uncover causative mutations, including de novo, new, and familial variants, related to epileptic syndromes and significantly enhance molecular diagnosis. The present study centres on the potential applications of next-generation exome sequencing in clinical diagnostics and the challenges encountered in the data processing of such data.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143966130","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
CRISPR/Cas9 Technology for Modifying Immune Checkpoint in CAR-T Cell Therapy for Hematopoietic Malignancies. CRISPR/Cas9技术修饰免疫检查点在CAR-T细胞治疗造血恶性肿瘤中的应用
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-04-14 DOI: 10.2174/0115665232357078250331180413
Forough Shams, Elham Sharif, Hajar Abbasi-Kenarsari, Nader Hashemi, Masoumeh Sadat Hosseini, Neda Heidari, Effat Noori, Ali Hossein Amini, Maryam Bazrgar, Maryam Rouhani, Yong Teng
{"title":"CRISPR/Cas9 Technology for Modifying Immune Checkpoint in CAR-T Cell Therapy for Hematopoietic Malignancies.","authors":"Forough Shams, Elham Sharif, Hajar Abbasi-Kenarsari, Nader Hashemi, Masoumeh Sadat Hosseini, Neda Heidari, Effat Noori, Ali Hossein Amini, Maryam Bazrgar, Maryam Rouhani, Yong Teng","doi":"10.2174/0115665232357078250331180413","DOIUrl":"https://doi.org/10.2174/0115665232357078250331180413","url":null,"abstract":"<p><p>Hematologic malignancies, which arise from dysregulation of hematopoiesis, are a group of cancers originating in cells with diminished capacity to differentiate into mature progeny and accumulating immature cells in blood-forming tissues such as lymph nodes and bone marrow. Immune- targeted therapies, such as Immune Checkpoint Blockade (ICB), chimeric antigen receptor T (CAR-T) cell therapy, and the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system, a precise, popular, and versatile genome engineering tool, have opened new avenues for the treatment of malignancies. Targeting immune checkpoints has revolutionized FDA approval in cytotoxic T-lymphocyte-associated protein 4 (CTLA-4), PD-1 (programmed death-1), and PDL1. According to the ICB and CAR techniques, the production of efficient CAR-T cells depends on the successful genetic modification of T cells, making them less susceptible to immune escape and suppression by cancer cells, which results in reduced off-target toxicity. Therefore, CRISPR/Cas9 has revolutionized the immune checkpoint-based approach for CAR-T cell therapy of hematologic malignancy. Continued research and clinical trials will undoubtedly pave the way for further advances in this field, ultimately benefiting patients and improving outcomes.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143970339","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Transforming RNA-Based Gene Therapy with Innovative Nanocarriers for siRNA and miRNA Delivery. 利用创新的纳米载体转化rna基因治疗,用于siRNA和miRNA的递送。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-04-14 DOI: 10.2174/0115665232355294250330002630
Jitendra Singh Chaudhary, Dilip Kumar Chanchal, Kuldeep Singh, Jeetendra Kumar Gupta, Divya Jain, Anubhav Dubey, Mahima Pandey, Shahbaz Khan
{"title":"Transforming RNA-Based Gene Therapy with Innovative Nanocarriers for siRNA and miRNA Delivery.","authors":"Jitendra Singh Chaudhary, Dilip Kumar Chanchal, Kuldeep Singh, Jeetendra Kumar Gupta, Divya Jain, Anubhav Dubey, Mahima Pandey, Shahbaz Khan","doi":"10.2174/0115665232355294250330002630","DOIUrl":"https://doi.org/10.2174/0115665232355294250330002630","url":null,"abstract":"<p><p>The cells have been given precise instructions proprio to the regulation of gene expression by the main genesis of Ryan-based gene therapy, which has revived cancer treatment and other disorders. The difficulty of delivering small interfering RNA (siRNA) and microRNA (miRNA) to a target cell is an enormous task and is often faced by researchers due to characteristic instabilities of these carriers and their poor uptake by the cell membrane. The new developments from nanocarrier technologies offer opportunities for better effectiveness of RNA therapy for its delivery and the effectiveness of the treatment regimen. The objective of this article is to provide an overview of the existing as well as the newest developments in nanocarrier technology, particularly as related to microRNA and small interfering RNA (siRNA) delivery. Their modes of operation and their uses in gene therapies are also examined as principles of their design. We focus on several nanocarrier technologies, which have shown proof of concept in multiple disciplines such as stability, controlled release profiles, and delivery. Lipid-based nanoparticles, polymeric systems, and hybrid nanocarriers are some of the platforms that fall under this category; however, this list is not exhaustive. We also study the idea that certain nanocarriers could have multiple functionalities, which would make it possible to improve cancer treatment by simultaneously carrying chemotherapy and genes. We aim to shed light on the future of RNA-based gene therapy by providing a thorough overview of recent research in the field. This will help us understand how novel nanocarrier technologies can tackle the delivery issues.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143968682","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Non-coding RNAs-based Therapy and Angiogenesis: A New Era for the Management of Gliomas. 基于非编码rna的治疗和血管生成:胶质瘤治疗的新时代。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-04-11 DOI: 10.2174/0115665232356703250325075309
Ozal Beylerli, Ilgiz Gareev, Elmar Musaev, Tatiana Ilyasova, Sergey Roumiantsev, Vladimir Chekhonin
{"title":"Non-coding RNAs-based Therapy and Angiogenesis: A New Era for the Management of Gliomas.","authors":"Ozal Beylerli, Ilgiz Gareev, Elmar Musaev, Tatiana Ilyasova, Sergey Roumiantsev, Vladimir Chekhonin","doi":"10.2174/0115665232356703250325075309","DOIUrl":"https://doi.org/10.2174/0115665232356703250325075309","url":null,"abstract":"<p><p>The relentless pursuit of understanding and combating glioblastoma (GBM), one of the most formidable foes in the realm of cancer, requires a deeper exploration of its intricate dynamics. Gliomas, particularly GBM, are known for their lethal nature, and a significant aspect of their pathogenesis lies in their ability to manipulate the blood vessels that sustain them. This complex relationship is governed by a multitude of molecular mechanisms involving a diverse array of cell types within the tumor microenvironment. Central to this intricate web of regulation are non-coding RNAs (ncRNAs), enigmatic molecules that have recently emerged as key players in cancer biology. These ncRNAs wield a remarkable influence on gene expression, often via epigenetic modifications and intricate control over angiogenesis-related molecules. Their role in GBM angiogenesis adds another layer of complexity to our understanding of this disease. In the realm of cancer therapeutics, targeting angiogenesis has become a prominent strategy. However, the efficacy of current antiangiogenic treatments against GBM is often transient, as these tumors can rapidly develop resistance, becoming even more aggressive. GBM employs a diverse set of strategies to foster its abnormal vasculature, which, in turn, holds the key to understanding why anti-angiogenic therapies often fall short of expectations. This review aims to shed light on potential strategies and novel perspectives to overcome GBM 's resistance to anti-angiogenic therapy. By exploring innovative approaches, including those centered on ncRNAs, we strive to chart a course toward more effective treatments. This journey into the depths of GBM 's complexities offers not only hope but also a blueprint for future research and therapeutic development. As we uncover the intricate mechanisms at play, we inch closer to the day when GBM is no longer an insurmountable adversary in the fight against cancer.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143979117","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Expression of miR-92a in Green Tea EGCG Preconditioned Adiposederived Stem Cells Ameliorates Inflammatory Response and Increases Cartilage Regeneration in RA Rats through KLF4/IL-17/MMP-2 Axis Modulation. 绿茶EGCG预处理脂肪源性干细胞中miR-92a的表达通过KLF4/IL-17/MMP-2轴调节改善RA大鼠炎症反应并增加软骨再生
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-04-03 DOI: 10.2174/0115665232339721250313075146
Tung-Sheng Chen, Wei-Wen Kuo, Chih-Yang Huang
{"title":"Expression of miR-92a in Green Tea EGCG Preconditioned Adiposederived Stem Cells Ameliorates Inflammatory Response and Increases Cartilage Regeneration in RA Rats through KLF4/IL-17/MMP-2 Axis Modulation.","authors":"Tung-Sheng Chen, Wei-Wen Kuo, Chih-Yang Huang","doi":"10.2174/0115665232339721250313075146","DOIUrl":"https://doi.org/10.2174/0115665232339721250313075146","url":null,"abstract":"<p><strong>Background: </strong>The global prevalence of rheumatoid arthritis (RA) is on the rise. Numerous studies have demonstrated the potential of stem cell-based therapies in RA treatment. Experimental evidence suggests that preconditioning enhances the regenerative capabilities of stem cells compared to their unconditioned counterparts.</p><p><strong>Objective: </strong>This study aimed to evaluate whether adipose-derived stem cells (ADSCs) preconditioned with green tea epigallocatechin gallate (EGCG) and miR-92a exhibit superior therapeutic effects in RA compared to unconditioned ADSCs.</p><p><strong>Methods: </strong>Both in vitro and in vivo models were employed. In the cellular model, ADSCs were preconditioned with EGCG and miR-92a. In the animal model, male Wistar rats were used, and RA was induced using the collagen-induced arthritis (CIA) model. Following RA induction, the animals were divided into six groups: Sham (healthy rats), RA (RA-induced rats), RA+ADSC (RA-induced rats receiving unconditioned ADSCs), RA+E-ADSC (RA-induced rats receiving EGCGpreconditioned ADSCs), RA+mic-ADSC (RA-induced rats receiving miR-92a mimicpreconditioned ADSCs), and RA+inh-ADSC (RA-induced rats receiving miR-92a inhibitorpreconditioned ADSCs).</p><p><strong>Results: </strong>In the cellular model, preconditioning with EGCG and miR-92a activated the CXCR4/p- Akt signaling pathway, thereby enhancing ADSC viability. In the animal model, RA induction caused several joint pathologies, including hind paw swelling, disrupted bone metabolism, immune cell infiltration, increased expression of IL-17, KLF4, and IL-6, as well as cartilage degradation. While transplantation of unconditioned ADSCs modestly improved these pathological features, the administration of E-ADSCs and mic-ADSCs significantly ameliorated these conditions in RA rats. Conversely, the therapeutic effects of E-ADSCs and mic-ADSCs were attenuated by the transplantation of inh-ADSCs.</p><p><strong>Conclusion: </strong>The therapeutic effects of E-ADSCs and mic-ADSCs in RA were strongly associated with the modulation of the KLF4/IL-17/MMP-2 axis. These findings suggest that ADSCs preconditioned with EGCG and miR-92a hold considerable clinical promise for the treatment of RA.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143779365","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Riding the Wave of Progress: Examining the Current Landscape and Future Potential of MicroRNAs in Cancer Gene Therapy. 乘着进步的浪潮:研究microrna在癌症基因治疗中的现状和未来潜力。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-03-24 DOI: 10.2174/0115665232353538250318075057
Saleem Ahmad, Hanif Khan, Njoka Irene Muthoni, Mohammed Alissa, Awaji Y Safhi, Fahad Y Sabei, Osama Abdulaziz, Khadijah Hassan Khan, Essam H Ibrahim, Safir Ullah Khan
{"title":"Riding the Wave of Progress: Examining the Current Landscape and Future Potential of MicroRNAs in Cancer Gene Therapy.","authors":"Saleem Ahmad, Hanif Khan, Njoka Irene Muthoni, Mohammed Alissa, Awaji Y Safhi, Fahad Y Sabei, Osama Abdulaziz, Khadijah Hassan Khan, Essam H Ibrahim, Safir Ullah Khan","doi":"10.2174/0115665232353538250318075057","DOIUrl":"10.2174/0115665232353538250318075057","url":null,"abstract":"<p><p>MicroRNAs, commonly referred to as miRNAs, exert a significant impact on cellular processes by coordinating post-transcriptional gene regulation. These non-coding RNAs, which are only 22 nucleotides long, form a part of the RNA-induced silencing complex (RISC) and play a crucial role in regulating gene expression. Their complex participation in cell proliferation, differentiation, and death highlights their crucial role in maintaining cellular balance. MicroRNAs have become significant contributors in the complex field of cancer biology, operating beyond the usual tasks of cells. Their dysregulation is closely intertwined with cancer initiation and development. miRNAs act as cellular regulators and regulate complex processes of gene expression. Disruption of this regulation can result in tumor development. This review article explores the intricate process of miRNA biosynthesis and its mechanisms, providing insights into its complex interactions with cancer. It also discusses the exciting field of miRNA-based cancer treatment. Exploring the therapeutic possibilities of these small RNA molecules presents opportunities for precision medicine, introducing a new age where miRNAs can be utilized to create targeted therapeutic interventions that mainly address the abnormal genetic characteristics that cause tumor formation. miRNAs provide a harmonious balance between understanding their biology and utilizing their therapeutic potential in cancer treatment. However, they also serve as conductors and possible therapeutic instruments in the symphony of molecular biology for gene therapy.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143699894","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
CRISPR-Cas9: Transforming Functional Genomics, Precision Medicine, and Drug Development - Opportunities, Challenges, and Future Directions. CRISPR-Cas9:转化功能基因组学,精准医学和药物开发-机遇,挑战和未来方向。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-03-21 DOI: 10.2174/0115665232376648250312050239
Zuber Khan, Mumtaz, Sumedha Gupta, Sidharth Mehan, Tarun Sharma, Manjeet Kumar, Pankaj Kumar Maurya, Arun Kumar Sharma, Ghanshyam Das Gupta, Acharan S Narula
{"title":"CRISPR-Cas9: Transforming Functional Genomics, Precision Medicine, and Drug Development - Opportunities, Challenges, and Future Directions.","authors":"Zuber Khan, Mumtaz, Sumedha Gupta, Sidharth Mehan, Tarun Sharma, Manjeet Kumar, Pankaj Kumar Maurya, Arun Kumar Sharma, Ghanshyam Das Gupta, Acharan S Narula","doi":"10.2174/0115665232376648250312050239","DOIUrl":"https://doi.org/10.2174/0115665232376648250312050239","url":null,"abstract":"<p><p>CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) is a groundbreaking gene-editing technology that enables scientists to make precise changes to the DNA of living organisms. It was first discovered in Escherichia coli and emerged as a breakthrough tool in molecular biology. This technique is essential because of its adaptability, affordability, and ease of use. It uses the adaptive immune response of bacteria and archaea to repel viral invasions. It significantly influences drug discovery, functional genomics, disease models, and pharmaceutical research. CRISPR-Cas9 is a better and more accurate way to change genes than other methods, such as zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). This technology promotes the generation of double-strand breaks in DNA, allowing for precise genetic alterations required for therapeutic target identification and confirmation. Functional genomics enables high-throughput screening (HTS) to identify gene functions, disease causes, and therapeutic targets. CRISPR-Cas9 increases drug development by enabling Cas9 to create novel antimicrobial drugs and cancer therapies. It has also helped to generate disease models, advance our understanding of neurodegenerative and other diseases, test a variety of chemicals, and facilitate precise genetic changes. Despite its promise, ethical considerations and the possibility of off-target effects require careful evaluation to ensure its safe and effective clinical application. This study investigates the current and future possibilities of CRISPR-Cas9 in drug development, focusing on its transformational influence and addressing the challenges and limitations of its therapeutic application.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143699892","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Polysaccharide-Based Delivery Systems for CRISPR/Cas Gene Therapy: Overcoming Challenges and Advancing Pharmaceutical Solutions. 基于多糖的CRISPR/Cas基因治疗递送系统:克服挑战和推进制药解决方案。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-03-10 DOI: 10.2174/0115665232369121250307075817
Deeksha Manchanda, Sunil Kumar, Manish Makhija
{"title":"Polysaccharide-Based Delivery Systems for CRISPR/Cas Gene Therapy: Overcoming Challenges and Advancing Pharmaceutical Solutions.","authors":"Deeksha Manchanda, Sunil Kumar, Manish Makhija","doi":"10.2174/0115665232369121250307075817","DOIUrl":"https://doi.org/10.2174/0115665232369121250307075817","url":null,"abstract":"<p><p>The advent of CRISPR/Cas gene-editing technology has revolutionized molecular biology, offering unprecedented precision and potential in treating genetic disorders, cancers, and other complex diseases. However, for CRISPR/Cas to be truly effective in clinical settings, one of the most significant challenges lies in the delivery of the CRISPR components, including guide RNA (gRNA) and Cas protein, into specific cells or tissues. Safe, targeted, and efficient delivery remains a critical bottleneck. Viral vectors, lipid nanoparticles, and synthetic polymers have been explored, but they come with limitations, such as immunogenicity, toxicity, and limited delivery capacity. Polysaccharide-based delivery systems, with their natural origin, biocompatibility, and versatile chemical properties, offer a promising alternative that could address these delivery challenges while advancing the pharmaceutical applications of CRISPR/Cas gene therapy.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143604026","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Antimicrobial Peptide Pipeline: A Bacteria-Centric AMP Predictor. 抗菌肽管道:以细菌为中心的AMP预测器。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-02-26 DOI: 10.2174/0115665232343790250120071445
Werner Pieter Veldsman, Qi Zhang, Qian Zhao, Lu Zhang, Zou Yuanjie
{"title":"The Antimicrobial Peptide Pipeline: A Bacteria-Centric AMP Predictor.","authors":"Werner Pieter Veldsman, Qi Zhang, Qian Zhao, Lu Zhang, Zou Yuanjie","doi":"10.2174/0115665232343790250120071445","DOIUrl":"https://doi.org/10.2174/0115665232343790250120071445","url":null,"abstract":"<p><strong>Introduction: </strong>Antimicrobial peptides (AMPs), unlike antibiotics, are encoded in genomes. AMPs are exported from the cell after expression and translation. In the case of bacteria, the exported peptides target other microbes to give the producing bacterium a competitive edge. While AMPs are sought after for their similar antimicrobial activity to traditional antibiotics, it is difficult to predict which combinations of amino acids will confer antimicrobial activity. Many computer algorithms have been designed to predict whether a sequence of amino acids will exhibit antimicrobial activity, but the vast majority of validated AMPs in databases are still of eukaryotic origin. This defies common sense since the vast majority of life on Earth is prokaryotic.</p><p><strong>Methods: </strong>The antimicrobial peptide pipeline, presented here, is a bacteria-centric AMP predictor that predicts AMPs by taking design inspiration from the sequence properties of bacterial genomes with the intention to improve the detection of naturally occurring bacterial AMPs. The pipeline integrates multiple concepts of comparative biology to search for candidate AMPs at the primary, secondary, and tertiary peptide structure levels.</p><p><strong>Results: </strong>Results showed that the antimicrobial peptide pipeline identifies known AMPs that are missed by state-of-the-art AMP predictors and that the pipeline yields more AMP candidates from real bacterial genomes than from fake genomes, with the rate of AMP detection being significantly higher in the genomes of six nosocomial pathogens than in the fake genomes.</p><p><strong>Conclusion: </strong>This bacteria-centric AMP pipeline enhances the detection of bacterial AMPs by incorporating sequence properties unique to bacterial genomes. It complements existing tools, addressing gaps in AMP detection and providing a promising avenue for discovering novel antimicrobial peptides.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143514883","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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