Current gene therapy最新文献_第9页

Duchenne Muscular Dystrophy Gene Therapy. 杜兴氏肌肉萎缩症基因疗法。

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-01-01 DOI: 10.2174/1566523223666221118160932

Fawzy A Saad, Jasen F Saad, Gabriele Siciliano, Luciano Merlini, Corrado Angelini

{"title":"Duchenne Muscular Dystrophy Gene Therapy.","authors":"Fawzy A Saad, Jasen F Saad, Gabriele Siciliano, Luciano Merlini, Corrado Angelini","doi":"10.2174/1566523223666221118160932","DOIUrl":"10.2174/1566523223666221118160932","url":null,"abstract":"Duchenne and Becker muscular dystrophies are allelic X-linked recessive neuromuscular diseases affecting both skeletal and cardiac muscles. Therefore, owing to their single X chromosome, the affected boys receive pathogenic gene mutations from their unknowing carrier mothers. Current pharmacological drugs are palliative that address the symptoms of the disease rather than the genetic cause imbedded in the Dystrophin gene DNA sequence. Therefore, alternative therapies like gene drugs that could address the genetic cause of the disease at its root are crucial, which include gene transfer/implantation, exon skipping, and gene editing. Presently, it is possible through genetic reprogramming to engineer AAV vectors to deliver certain therapeutic cargos specifically to muscle or other organs regardless of their serotype. Similarly, it is possible to direct the biogenesis of exosomes to carry gene editing constituents or certain therapeutic cargos to specific tissue or cell type like brain and muscle. While autologous exosomes are immunologically inert, it is possible to camouflage AAV capsids, and lipid nanoparticles to evade the immune system recognition. In this review, we highlight current opportunities for Duchenne muscular dystrophy gene therapy, which has been known thus far as an incurable genetic disease. This article is a part of Gene Therapy of Rare Genetic Diseases thematic issue.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"17-28"},"PeriodicalIF":3.6,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9328449","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

Exosome miR-30a-5p Regulates Glomerular Endothelial Cells' EndMT and Angiogenesis by Modulating Notch1/VEGF Signaling Pathway. 外显子miR-30a-5p通过调节Notch1/VEGF信号通路调节肾小球内皮细胞的EndMT和血管生成。

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-01-01 DOI: 10.2174/0115665232258527230919071328

Yaxian Ning, Xiaochun Zhou, Gouqin Wang, Lili Zhang, Jianqin Wang

{"title":"Exosome miR-30a-5p Regulates Glomerular Endothelial Cells' EndMT and Angiogenesis by Modulating Notch1/VEGF Signaling Pathway.","authors":"Yaxian Ning, Xiaochun Zhou, Gouqin Wang, Lili Zhang, Jianqin Wang","doi":"10.2174/0115665232258527230919071328","DOIUrl":"10.2174/0115665232258527230919071328","url":null,"abstract":"Background: Diabetic nephropathy (DN) is one of the microvascular complications of diabetes. Endothelial-mesenchymal transition (EndMT) and endothelial damage lead to abnormal angiogenesis in DN.Objectives: This study aimed to investigate the role of exosome miR-30a-5p in high glucose (HG)-induced glomerular endothelial cells (GECs) dysfunction and explore the underlying mechanisms.Methods: GECs were cultured in normal glucose (5.5 mM) and HG (30 mM) conditions. The recipient GECs were transfected with exosome or miR-30a-5p mimic/inhibitor and then detected by using CCK-8 and flow cytometry assay. Luciferase analysis was used to verify miR-30a-5p acted on notch homolog protein 1 (Notch1). RT-qPCR and Western blot were used to detect the expression of VE-cadherin, α-SMA, vascular endothelial growth factor (VEGF) and Notch1. In vivo, exosome miR-30a-5p was administered to DN mice, and periodic acid-Schiff (PAS) staining, UTP levels, and HbA1c levels were measured.Results: The expression of miR-30a-5p was downregulated in HG-treated GECs. Exosome miR-30a-5p significantly promoted cell proliferation, and migration and reduced apoptosis of GECs under HG conditions. MiR-30a-5p directly targeted the 3-UTR region of Notch1. Exosome miR-30a-5p reduced the expression levels of Notch1 and VEGF, both at mRNA and protein levels. Furthermore, exosome miR-30a-5p inhibited HG-induced EndMT, as evidenced by increased VE-cadherin and reduced α-SMA. In vivo studies demonstrated that exosome miR-30a-5p reduced serum HbA1c levels and 24-hour urine protein quantification.Conclusion: This study provides evidence that exosome miR-30a-5p suppresses EndMT and abnormal angiogenesis of GECs by modulating the Notch1/VEGF signaling pathway. These findings suggest that exosome miR-30a-5p could be a potential therapeutic strategy for the treatment of DN.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"159-177"},"PeriodicalIF":3.6,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41106454","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

Gene Therapy for Rare Genetic Diseases. 罕见遗传病的基因疗法。

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-01-01 DOI: 10.2174/1566523223666230320120839

Kenneth Lundstrom, Evangelia Yannaki, Janice Chou, Fawzy A Saad

引用次数: 0

FIGNL1 Promotes Hepatocellular Carcinoma Formation via Remodeling ECM-receptor Interaction Pathway Mediated by HMMR. FIGNL1通过HMMR介导的重塑ECM受体相互作用途径促进肝细胞癌的形成。

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-01-01 DOI: 10.2174/0115665232274223231017052707

Jiabei Wang, Linmao Sun, Yao Liu, Yunguang Zhang

{"title":"FIGNL1 Promotes Hepatocellular Carcinoma Formation via Remodeling ECM-receptor Interaction Pathway Mediated by HMMR.","authors":"Jiabei Wang, Linmao Sun, Yao Liu, Yunguang Zhang","doi":"10.2174/0115665232274223231017052707","DOIUrl":"10.2174/0115665232274223231017052707","url":null,"abstract":"Background: The development of novel biomarkers is crucial for the treatment of HCC. In this study, we investigated a new molecular therapeutic target for HCC. Fidgetin-like 1 (FIGNL1) has been reported to play a vital role in lung adenocarcinoma. However, the potential function of FIGNL1 in HCC is still unknown.Objective: This study aims to investigate the key regulatory mechanisms of FIGNL1 in the formation of HCC.Methods: The regulatory effect of FIGNL1 on HCC was studied by lentivirus infection. In vitro, the effects of FIGNL1 on the proliferation, migration and apoptosis of cells were investigated by CCK8, colony formation assay, transwell and flow cytometry. Meanwhile, the regulation of FIGNL1 on HCC formation in vivo was studied by subcutaneous transplanted tumors. In addition, using transcriptome sequencing technology, we further explored the specific molecular mechanism of FIGNL1 regulating the formation of HCC.Results: Functionally, we demonstrated that FIGNL1 knockdown significantly inhibited HCC cell proliferation, migration and promoted cell apoptosis in vitro. Similarly, the knockdown of FIGNL1 meaningfully weakened hepatocarcinogenesis in nude mice. Transcriptome sequencing revealed that FIGNL1 affected the expression of genes involved in extracellular matrix-receptor (ECM-receptor) interaction pathway, such as hyaluronan mediated motility receptor (HMMR). Further validation found that overexpression of HMMR based on knockdown FIGNL1 can rescue the expression abundance of related genes involved in the ECM-receptor interaction pathway.Conclusion: Our study revealed that FIGNL1 could modulate the ECM-receptor interaction pathway through the regulation of HMMR, thus regulating the formation of HCC.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"249-263"},"PeriodicalIF":3.6,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11071652/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71479127","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy. 用 AAV 治疗血友病：探索基因疗法的前沿。

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-01-01 DOI: 10.2174/0115665232279893231228065540

Safir Ullah Khan, Munir Ullah Khan, Muhammad Suleman, Amrah Inam, Muhammad Azhar Ud Din

{"title":"Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy.","authors":"Safir Ullah Khan, Munir Ullah Khan, Muhammad Suleman, Amrah Inam, Muhammad Azhar Ud Din","doi":"10.2174/0115665232279893231228065540","DOIUrl":"10.2174/0115665232279893231228065540","url":null,"abstract":"Gene therapy for hemophilia has advanced tremendously after thirty years of continual study and development. Advancements in medical science have facilitated attaining normal levels of Factor VIII (FVIII) or Factor IX (FIX) in individuals with haemophilia, thereby offering the potential for their complete recovery. Despite the notable advancements in various countries, there is significant scope for further enhancement in haemophilia gene therapy. Adeno-associated virus (AAV) currently serves as the primary vehicle for gene therapy in clinical trials targeting haemophilia. Subsequent investigations will prioritize enhancing viral capsid structures, transgene compositions, and promoters to achieve heightened transduction efficacy, diminished immunogenicity, and more predictable therapeutic results. The present study indicates that whereas animal models have transduction efficiency that is over 100% high, human hepatocytes are unable to express clotting factors and transduction efficiency to comparable levels. According to the current study, achieving high transduction efficiency and high levels of clotting factor expression in human hepatocytes is still insufficient. It is also crucial to reduce the risk of cellular stress caused by protein overload. Despite encountering various hurdles, the field of haemophilia gene therapy holds promise for the future. As technology continues to advance and mature, it is anticipated that a personalized therapeutic approach will be developed to cure haemophilia effectively.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"265-277"},"PeriodicalIF":3.6,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139570019","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

Segmentation of Thoracic Organs through Distributed Extraction of Visual Feature Patterns Utilizing Resio-Inception U-Net and Deep Cluster Recognition Techniques. 利用 Resio-Inception U-Net 和深度聚类识别技术，通过分布式提取视觉特征模式分割胸腔脏器

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-01-01 DOI: 10.2174/0115665232262165231201113932

Karthikeyan Saminathan, Tathagat Banerjee, Devi Priya Rangasamy, Meenalosini Vimal Cruz

{"title":"Segmentation of Thoracic Organs through Distributed Extraction of Visual Feature Patterns Utilizing Resio-Inception U-Net and Deep Cluster Recognition Techniques.","authors":"Karthikeyan Saminathan, Tathagat Banerjee, Devi Priya Rangasamy, Meenalosini Vimal Cruz","doi":"10.2174/0115665232262165231201113932","DOIUrl":"10.2174/0115665232262165231201113932","url":null,"abstract":"Background: Segmentation of medical images plays a key role in the correct identification and management of different diseases. In this study, we present a new segmentation method that meets the difficulties posed by sophisticated organ shapes in computed tomography (CT) images, particularly targeting lung, breast, and gastric cancers.Methods: Our suggested methods, Resio-Inception U-Net and Deep Cluster Recognition (RIUDCR), use a Residual Inception Architecture, which combines the power of residual connections and inception blocks to achieve cutting-edge segmentation performance while reducing the risk of overfitting.Results: We present mathematical equations and functions that describe the design, including the encoding and decoding steps within the UC-Net system. Furthermore, we provide strong testing results that show the effectiveness of our method. Through thorough testing on varied datasets, our method regularly beats current techniques, achieving amazing precision and stability in organ task segmentation. These results show the promise of our residual inception architecture in better medical picture analysis.Conclusion: In summary, our research not only shows a state-of-the-art segment methodology but also reinforces its usefulness through thorough testing. The inclusion of residual inception architecture in medical picture segmentation offers good possibilities for improving the identification and management of disease planning.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"217-238"},"PeriodicalIF":3.6,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139680756","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

Splicing DNA Damage Adaptations for the Management of Cancer Cells 剪接DNA损伤对癌细胞管理的适应性

IF 3.6 4区医学

Current gene therapy Pub Date : 2023-11-29 DOI: 10.2174/0115665232258528231018113410

Arun Kumar Singh, Deepika Yadav, Rishabha Malviya

引用次数: 0

Acknowledgements to Reviewers 审稿人致谢

4区医学

Current gene therapy Pub Date : 2023-10-01 DOI: 10.2174/156652322305230816165521

引用次数: 0

Meet the Editorial Board Member 与编辑委员会成员见面

4区医学

Current gene therapy Pub Date : 2023-02-01 DOI: 10.2174/156652322301221113224455

Lei Chen

引用次数: 0

Effective Combination of Single Cell Analysis and Gene Therapy 单细胞分析与基因治疗的有效结合

IF 3.6 4区医学

Current gene therapy Pub Date : 2023-02-01 DOI: 10.2174/156652322301221113224009

Liang Cheng

引用次数: 0