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In vivo gene therapy: A strategy for mutations, degenerations, and tumors 体内基因治疗:一种治疗突变、变性和肿瘤的策略
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-08-19 DOI: 10.1016/j.gendis.2025.101808
Tao Wang , Mingyang Yu , Ping Liu , Zhiqiang Song , Cheng Li , Jianmin Yang , Na Liu
{"title":"In vivo gene therapy: A strategy for mutations, degenerations, and tumors","authors":"Tao Wang ,&nbsp;Mingyang Yu ,&nbsp;Ping Liu ,&nbsp;Zhiqiang Song ,&nbsp;Cheng Li ,&nbsp;Jianmin Yang ,&nbsp;Na Liu","doi":"10.1016/j.gendis.2025.101808","DOIUrl":"10.1016/j.gendis.2025.101808","url":null,"abstract":"<div><div>Gene mutations, organ function degeneration, and carcinogenesis are the primary threats to human health. Gene therapy, which involves the addition, deletion, regulation, and editing of genes, as well as the development of genetic vaccines, can potentially cure genetic mutation disorders, degenerative diseases, and cancers. <em>Ex vivo</em> gene therapy has recently been used to treat monogenetic mutation diseases of the hematopoietic system and cancers. However, <em>in vivo</em> gene therapy remains inapplicable. The primary elements of <em>in vivo</em> gene therapy include deoxyribonucleic acid (DNA) nucleases (<em>e.g.</em>, zinc finger nucleases, transcription activator-like effector nucleases), CRISPR-Cas system, base editors, prime editors, and delivery vectors (<em>e.g.</em>, viral and non-viral vehicles). According to the development of DNA nucleases and delivery vectors, <em>in vivo</em> gene therapy can be made available for future clinical use. The current review summarizes the development of DNA nucleases and delivery vectors for <em>in vivo</em> gene therapy, emphasizing recent progress.</div></div>","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101808"},"PeriodicalIF":9.4,"publicationDate":"2025-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145266720","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Magnolol mitigates morphine-induced analgesic tolerance via EGFR-mediated inhibition of microglial activation and neuroinflammatory reduction 厚朴酚通过egfr介导的小胶质细胞激活抑制和神经炎症减少来减轻吗啡诱导的镇痛耐受
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-08-15 DOI: 10.1016/j.gendis.2025.101802
Weixin Lin , Yuxuan Wang , Qisheng Wang , Jiamin Huang , Ziting Zhou , Yongwei Jiang , Fenfen Qin , Zhonghao Li , Hui Wang , Zijing Wang , Haotian Pan , Qian Wang , Shanzhong Tan , Zhigang Lu
{"title":"Magnolol mitigates morphine-induced analgesic tolerance via EGFR-mediated inhibition of microglial activation and neuroinflammatory reduction","authors":"Weixin Lin ,&nbsp;Yuxuan Wang ,&nbsp;Qisheng Wang ,&nbsp;Jiamin Huang ,&nbsp;Ziting Zhou ,&nbsp;Yongwei Jiang ,&nbsp;Fenfen Qin ,&nbsp;Zhonghao Li ,&nbsp;Hui Wang ,&nbsp;Zijing Wang ,&nbsp;Haotian Pan ,&nbsp;Qian Wang ,&nbsp;Shanzhong Tan ,&nbsp;Zhigang Lu","doi":"10.1016/j.gendis.2025.101802","DOIUrl":"10.1016/j.gendis.2025.101802","url":null,"abstract":"","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101802"},"PeriodicalIF":9.4,"publicationDate":"2025-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145061127","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Carnosine Dipeptidase(Cndp): An emerging therapeutic target for metabolic diseases and cancers 肌肽二肽酶(Cndp):代谢性疾病和癌症的新靶点
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-08-13 DOI: 10.1016/j.gendis.2025.101804
Liang Zhou , Shuxia Zhang , Yunqi Zhang , Yun Luo , Xiaobo Sun
{"title":"Carnosine Dipeptidase(Cndp): An emerging therapeutic target for metabolic diseases and cancers","authors":"Liang Zhou ,&nbsp;Shuxia Zhang ,&nbsp;Yunqi Zhang ,&nbsp;Yun Luo ,&nbsp;Xiaobo Sun","doi":"10.1016/j.gendis.2025.101804","DOIUrl":"10.1016/j.gendis.2025.101804","url":null,"abstract":"<div><div>Metabolic diseases, associated with high morbidity and mortality rates, pose a challenge to global public health and a significant burden on society. Since the discovery of carnosinase-2(CNDP2)-mediated synthesis of lactate and phenylalanine, which subsequently forms N-Lactoyl-Phenylalanine (Lac-Phe) to inhibit food intake and obesity, the carnosine dipeptidases (CNDPs)have attracted increasing scientific interest. Although the role of CNDP in diabetic nephropathy has been extensively studied, its role in other metabolic diseases remains unclear. In this study, we have overviewed the enzymatic and other roles of CNDP proteins focusing on recent research demonstrating the regulatory roles of CNDP on various metabolic diseases. Increasing evidence indicates that carnosinase-1(CNDP1) and carnosinase-2 are crucial for the management of metabolic diseases under both physiological and pathological conditions. Moreover, interest in the pharmacological modulators of CNDP has been steadily increasing. Overall, we suggest that CNDP can be considered a promising therapeutic target for the effective treatment of metabolic diseases.</div></div>","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101804"},"PeriodicalIF":9.4,"publicationDate":"2025-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145217169","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Mitochondria and tumorigenesis: Molecular basis and therapeutic implications 线粒体和肿瘤发生:分子基础和治疗意义
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-08-12 DOI: 10.1016/j.gendis.2025.101806
Chen Huang , Zichuan Xie , Jiajin Li , Chenliang Zhang
{"title":"Mitochondria and tumorigenesis: Molecular basis and therapeutic implications","authors":"Chen Huang ,&nbsp;Zichuan Xie ,&nbsp;Jiajin Li ,&nbsp;Chenliang Zhang","doi":"10.1016/j.gendis.2025.101806","DOIUrl":"10.1016/j.gendis.2025.101806","url":null,"abstract":"<div><div>Mitochondria, vital organelles within cells, govern energy metabolism. They play a pivotal role in maintaining redox homeostasis and are instrumental in the initiation and transmission of cell death signals, along with the synthesis of biological macromolecules. The role of mitochondria in tumor evolution and treatment has recently been the focus of extensive research. Studies indicate that the quality and biogenesis of mitochondria, along with their structure, functions, and macromolecule synthesis relevant to it, are intimately linked to tumorigenesis and the prognostic outcomes of clinical treatments. As such, therapies targeting mitochondria offer promising avenues to augment the efficacy of tumor treatment. We summarized the inherent links between mitochondrial structure, mitochondrial genes, metabolism of mitochondrial-related biological macromolecules, and mitochondria-regulated cell death in relation to tumorigenesis and progression. Furthermore, we reviewed the latest research progress in targeting mitochondria for tumor therapy. This study suggests that targeting mitochondria could open new avenues for developing tumor therapy.</div></div>","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101806"},"PeriodicalIF":9.4,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145155776","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Protein arginine methyltransferase 5 as a novel therapeutic target in solid tumors 蛋白精氨酸甲基转移酶5作为实体瘤的新治疗靶点
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-08-06 DOI: 10.1016/j.gendis.2025.101796
Ping Song , Fan Yang
{"title":"Protein arginine methyltransferase 5 as a novel therapeutic target in solid tumors","authors":"Ping Song ,&nbsp;Fan Yang","doi":"10.1016/j.gendis.2025.101796","DOIUrl":"10.1016/j.gendis.2025.101796","url":null,"abstract":"<div><div>Protein arginine methyltransferase 5 (PRMT5) is the primary type II methyltransferase that mainly catalyzes symmetric demethylation of arginine residues in both histone and nonhistone proteins. Increasing evidence has demonstrated that PRMT5 is indispensable in tumorigenesis and acquired therapeutic resistance in multiple malignancies. This review summarizes the clinical significance of PRMT5 in solid tumors such as lung cancer, breast cancer, and glioblastoma, its role in tumor immunology, and current clinical trials of PRMT5 inhibitors, and discusses the clinical status, current dilemma, and future perspectives of PRMT5 inhibition as a novel therapeutic strategy.</div></div>","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101796"},"PeriodicalIF":9.4,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145217178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Genetic characteristics and prognosis of m6A RNA methylation regulator in acute myeloid leukemia 急性髓性白血病中m6A RNA甲基化调控因子的遗传特征及预后
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-08-05 DOI: 10.1016/j.gendis.2025.101789
Kaili Liao , Daixin Guo , Yujie Hu , Jiarong Wen , Yuhan Xu , Xinyi Bai , Jinting Cheng , Beining Zhang , Xiaozhong Wang
{"title":"Genetic characteristics and prognosis of m6A RNA methylation regulator in acute myeloid leukemia","authors":"Kaili Liao ,&nbsp;Daixin Guo ,&nbsp;Yujie Hu ,&nbsp;Jiarong Wen ,&nbsp;Yuhan Xu ,&nbsp;Xinyi Bai ,&nbsp;Jinting Cheng ,&nbsp;Beining Zhang ,&nbsp;Xiaozhong Wang","doi":"10.1016/j.gendis.2025.101789","DOIUrl":"10.1016/j.gendis.2025.101789","url":null,"abstract":"","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101789"},"PeriodicalIF":9.4,"publicationDate":"2025-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145044114","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Unveiling the roles of SPP1+ macrophage and IGFBP2+ fibroblast in lung adenosquamous carcinoma through single-cell analysis 通过单细胞分析揭示SPP1+巨噬细胞和IGFBP2+成纤维细胞在肺腺鳞癌中的作用
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-07-24 DOI: 10.1016/j.gendis.2025.101779
Yao Lin , Yaxin Chen , Dandan Xiong , Jing Huang , Hongmo Liu , Yawen Qi , Jinfeng Chen , Jun Meng , Yueqi Li , Jingyuan Yang , Yi Bao , Wenxing Li , Li Yang , Sanqi An
{"title":"Unveiling the roles of SPP1+ macrophage and IGFBP2+ fibroblast in lung adenosquamous carcinoma through single-cell analysis","authors":"Yao Lin ,&nbsp;Yaxin Chen ,&nbsp;Dandan Xiong ,&nbsp;Jing Huang ,&nbsp;Hongmo Liu ,&nbsp;Yawen Qi ,&nbsp;Jinfeng Chen ,&nbsp;Jun Meng ,&nbsp;Yueqi Li ,&nbsp;Jingyuan Yang ,&nbsp;Yi Bao ,&nbsp;Wenxing Li ,&nbsp;Li Yang ,&nbsp;Sanqi An","doi":"10.1016/j.gendis.2025.101779","DOIUrl":"10.1016/j.gendis.2025.101779","url":null,"abstract":"","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101779"},"PeriodicalIF":9.4,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145046867","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Polymorphisms of mismatch repair pathway genes predict clinical outcomes in acute myeloid leukemia patients 错配修复途径基因多态性预测急性髓性白血病患者的临床预后
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-07-16 DOI: 10.1016/j.gendis.2025.101774
Amin Zhang , Wancheng Liu , Can Can , Xiaodong Guo , Hexiao Jia , Yihong Wei , Hanyang Wu , Chunyan Ji , Daoxin Ma
{"title":"Polymorphisms of mismatch repair pathway genes predict clinical outcomes in acute myeloid leukemia patients","authors":"Amin Zhang ,&nbsp;Wancheng Liu ,&nbsp;Can Can ,&nbsp;Xiaodong Guo ,&nbsp;Hexiao Jia ,&nbsp;Yihong Wei ,&nbsp;Hanyang Wu ,&nbsp;Chunyan Ji ,&nbsp;Daoxin Ma","doi":"10.1016/j.gendis.2025.101774","DOIUrl":"10.1016/j.gendis.2025.101774","url":null,"abstract":"","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101774"},"PeriodicalIF":9.4,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145044115","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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 regulatory role of CASZ1 in keratinocyte differentiation and skin barrier function in atopic dermatitis CASZ1在特应性皮炎中角化细胞分化和皮肤屏障功能中的调节作用
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-07-11 DOI: 10.1016/j.gendis.2025.101767
Xuemei Li , Yuqiong Huang , Changdeok Kim , Li Fu , Hongxiang Chen , Kyungeun Jung
{"title":"The regulatory role of CASZ1 in keratinocyte differentiation and skin barrier function in atopic dermatitis","authors":"Xuemei Li ,&nbsp;Yuqiong Huang ,&nbsp;Changdeok Kim ,&nbsp;Li Fu ,&nbsp;Hongxiang Chen ,&nbsp;Kyungeun Jung","doi":"10.1016/j.gendis.2025.101767","DOIUrl":"10.1016/j.gendis.2025.101767","url":null,"abstract":"","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101767"},"PeriodicalIF":9.4,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145060425","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Hemophagocytic lymphohistiocytosis: Unraveling the role of SARS-CoV-2 infection 噬血细胞淋巴组织细胞病:揭示SARS-CoV-2感染的作用
IF 9.4 2区 医学
Genes & Diseases Pub Date : 2025-07-09 DOI: 10.1016/j.gendis.2025.101764
Erin Murphy, Krzysztof Data, Dominika Domagała, Julia Niebora, Artur Bryja, Małgorzata Józkowiak, Hanna Piotrowska-Kempisty, Piotr Dziegiel, Bartosz Kempisty, Paul Mozdziak
{"title":"Hemophagocytic lymphohistiocytosis: Unraveling the role of SARS-CoV-2 infection","authors":"Erin Murphy,&nbsp;Krzysztof Data,&nbsp;Dominika Domagała,&nbsp;Julia Niebora,&nbsp;Artur Bryja,&nbsp;Małgorzata Józkowiak,&nbsp;Hanna Piotrowska-Kempisty,&nbsp;Piotr Dziegiel,&nbsp;Bartosz Kempisty,&nbsp;Paul Mozdziak","doi":"10.1016/j.gendis.2025.101764","DOIUrl":"10.1016/j.gendis.2025.101764","url":null,"abstract":"","PeriodicalId":12689,"journal":{"name":"Genes & Diseases","volume":"13 1","pages":"Article 101764"},"PeriodicalIF":9.4,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144997815","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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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