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Emerging Therapeutics

Section edited by Gennaro Ciliberto

© Parilov / stock.adobe.comThe Emerging Therapeutics section is dedicated to the wave of emerging therapeutics arising from the intense research efforts and therapeutic breakthroughs based on recent scientific and technological developments. 

The section is interested in original papers, reviews and perspectives on the advancements in the field, including genetic vaccines, monoclonal antibodies, CAR-Ts, gene therapy with viral vectors, clinical applications of CRISPR/CAS9 gene editing, microbiota-modulating approaches, ‘omics approaches, emerging therapeutics from the development of new technologies for drug development, and discovery of new intervention targets and the possibility for new drug combinations.

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  1. Tongue squamous cell carcinoma (TSCC) is a common oral cancer that has a high propensity for recurrence and metastasis. Therefore, TSCC has a 50% 5-year survival rate. Platinum-based chemotherapy is an effecti...

    Authors: Chunyan Li, Wen Peng, Zhaoming Zhong, Chao Zhang, Xuemin Wang, Rujia Qin, Qian Lei, Jialing Lv, Feineng Liu, Yinghong Zhao, Zengbo Lv, Chongxin Li, Shixuan Yang, Haixia Zhang, Zhi Tao and Chuanzheng Sun
    Citation: Journal of Translational Medicine 2025 23:1142
  2. Aberrant upregulation of RNA polymerase I (Pol I) transcription and its dedicated machinery plays a pivotal role in tumor progression and chemoresistance. RRN3, a Pol I-specific transcription initiation factor...

    Authors: Shashanka Shekhar Sarkar, Mansi Sharma, Arpita Karmakar, Kousar Jahan, Sheetanshu Saproo, Soumojit Biswas, Sakshi Nautiyal, Ipsita Roy, P. V. Bharatam and Srivatsava Naidu
    Citation: Journal of Translational Medicine 2025 23:1131
  3. Human Papillomavirus-associated cancer remains a global health issue despite the availability of prophylactic vaccination. Two single-chain recombinant antibodies specific for the high-risk HPV16-E6 and E7 onc...

    Authors: Susanna Falcucci, Sabrina Tait, Maria Vincenza Chiantore, Anna Rita Ciccaglione, Carla Amici, Paola Di Bonito and Luisa Accardi
    Citation: Journal of Translational Medicine 2025 23:1095
  4. Triple-negative breast cancer (TNBC) is an aggressive malignancy with limited therapeutic options. Immune checkpoint inhibitors targeting the programmed death-ligand 1 (PD-L1) pathway show restricted efficacy ...

    Authors: Liying Song, Sumin Tang, Xuelei Pi, Yuanyuan Yan, Chenxi Hu, Liang Liu, Hongna Chen, Yating Zhang, Shishi Liu, Dan Yu, Chengkai Yin, Tianyan Liu, Xu Li, Deshan Li, Zhenzhong Wang, Wei Zhu…
    Citation: Journal of Translational Medicine 2025 23:1056
  5. Prostate cancer (PCa) remains the most prevalent malignancy among men, with the inevitable emergence of castration-resistant prostate cancer (CRPC) presenting the greatest challenge. Accumulating evidence has ...

    Authors: Hongtao Xu, Yifan Hou, Zhenhua Zhao, Jianhua Zhang, Pan Li, Yujia Cao, Xiaobo Nie and Junqing Hou
    Citation: Journal of Translational Medicine 2025 23:1045
  6. Hepatocellular carcinoma (HCC) is a primary liver malignancy characterized by a high mortality rate and unfavorable prognosis. Altered epigenetic modifications have been closely associated with cancer developm...

    Authors: Yang Xu, Fei Lan, Chenguang Yang and Pengfei Li
    Citation: Journal of Translational Medicine 2025 23:1022
  7. Anti-angiogenic therapy is a clinically validated method for cancer treatment. It was previously revealed that concurrent targeting of angiogenic and death receptor signaling pathways by a multivalent DR5-spec...

    Authors: Irina N. Druzhkova, Anna G. Orlova, Anastasiia S. Fedulova, Arina V. Avakiants, Alina A. Isakova, Ekaterina V. Kukovyakina, Yuan Zijian, Ekaterina A. Plotnikova, Galina V. Trunova, Andrey A. Pankratov, Anton A. Plekhanov, Alexey A. Kurnikov, Pavel V. Subochev, Alexey K. Shaytan, Marine E. Gasparian, Mikhail P. Kirpichnikov…
    Citation: Journal of Translational Medicine 2025 23:949
  8. Small cell lung cancer (SCLC) remains a difficult disease to treat with poor long-term survival rates. New therapies offer modest overall survival benefit beyond that of chemotherapy alone, necessitating the d...

    Authors: Bryony Heath, Bubacarr G. Kaira, Dhruma Thakker, Omar J. Mohammed, Ruhul Choudhury, Foram Dave, Poonam Vaghela, Elena Dubinina, Tina Parsons, Lindy Durrant and Mireille Vankemmelbeke
    Citation: Journal of Translational Medicine 2025 23:940
  9. Exosomes are extracellular vesicles of nanoscale, which represent the characteristics of the cells of origin. Tumor cell-derived exosomes (TEXs) obtain the characteristics of the tumor cells, which meanwhile t...

    Authors: Mengyue Li, Yifan Wang, Hu Zhang, Xueqing Wang, Lin He and Jingying Dai
    Citation: Journal of Translational Medicine 2025 23:925
  10. R-loop is a special DNA–RNA hybrid genomic structure. Since its identification, the molecular mechanisms of physiological processes such as class switch recombination have been revealed, uncontrolled regulatio...

    Authors: Yuan Sun, Sheng Wang, Nan Ge, Jintao Guo, Guoxin Wang, Fan Yang and Siyu Sun
    Citation: Journal of Translational Medicine 2025 23:912
  11. Osteosarcoma is the most common primary malignant bone tumor affecting children and young adults. Metastatic osteosarcoma has poor prognosis and represents a significant unmet medical need in clinical settings...

    Authors: Cheng Dai, Bin Shen, Shenyan Liu, Cong Li, Shuqun Yang, Jie Wang, Jie Zhang, Manqi Liu, Zhixuan Zhu, Wan Shi, Qi Zhang, Zhui Chen and Nannan Zhang
    Citation: Journal of Translational Medicine 2025 23:900

    The Correction to this article has been published in Journal of Translational Medicine 2025 23:1063

  12. C/EBPα (CCAAT enhancer binding protein) is a pivotal transcription factor with widespread expression in multiple tissues, including hematopoietic tissues, liver, lungs, mammary glands, skin, and adipose tissue...

    Authors: Xin Li, Tengkun Dai, Jiamin Zhao, Yidan Liang, Longqing Chen, Chao Chen, Mengmeng Guo, Yong Han, Lin Xu and Juanjuan Zhao
    Citation: Journal of Translational Medicine 2025 23:894
  13. Polystyrene (PS) particles, which have been recognized as emerging environmental pollutants, have increasingly been associated with various human diseases. However, their specific role and underlying mechanism...

    Authors: Shengming Lu, Ruipeng Wu, Weijian Li, Zhenyu Fu, Xuefei Ding, Yang Luan, Huang Tianbao and Yuhua Huang
    Citation: Journal of Translational Medicine 2025 23:891
  14. Gastrointestinal (GI) cancer is a series of malignant cancer mainly affecting the GI tract. Chemotherapy is one of the most treatment strategies used in GI cancer treatment, especially in advanced cases. Howev...

    Authors: Chenhe Li, Shunjia Xing, Dechun Zhang, Ruoyu Li, Qian Li, Hongliang Luo and Fangteng Liu
    Citation: Journal of Translational Medicine 2025 23:889
  15. Triple-negative breast cancer (TNBC) is an aggressive subtype lacking targetable proteins for treatment. PARP inhibitors (PARPi) are effective in BRCA-mutated cancers but have limited utility in non-germline BRCA...

    Authors: Ho Tsoi, George Man Hong Leung, Ellen Pui Sum Man, Chan Ping You, Koei Ho Lam Cheung, Kelvin Yuen Kwong Chan, Chun Gong, Michael Shing Yan Huen and Ui Soon Khoo
    Citation: Journal of Translational Medicine 2025 23:860
  16. Several antibody–drug conjugates (ADC) targeting Trophoblast cell surface antigen 2 (Trop-2) have been developed, demonstrating significant therapeutic efficacy in triple-negative breast cancer and non-small c...

    Authors: Xiaoying Qian, Chuanhong Luo, Chen Fang, Yongbin Wu, Weiwei Hong, Biao Yu, Guizhen Qin, Yan Yin, Xinyuan Yao, Xin Ye, Bingbiao Zhou, Chengsi Shu, Dengying Chen, Zhaoqing Li, Shanshan Wang, Yong Wang…
    Citation: Journal of Translational Medicine 2025 23:853
  17. Esophageal cancer is the 11th most common malignancy and the 7th leading cause of cancer-related death globally. Identifying key molecules and underlying mechanisms in the progression of esophageal cancer repr...

    Authors: Xiaochao Ma, Yulu He, Yue Yang, Tianyu Lu, Ze Tang, Youbin Cui and Rui Wang
    Citation: Journal of Translational Medicine 2025 23:562
  18. The molecular landscape of lung neuroendocrine neoplasms is still poorly characterized, making it difficult to develop a molecular classification and personalized therapeutic approaches. Significant clinical h...

    Authors: Giulia Pecora, Camilla Mancini, Rossella Mazzilli, Virginia Zamponi, Stefano Telese, Stefano Scalera, Marcello Maugeri-Saccà, Ludovica Ciuffreda, Francesca De Nicola, Maurizio Fanciulli, Anna La Salvia, Massimiliano Mancini, Andrea Vecchione, Alessandra Siciliani, Mohsen Ibrahim, Diana Bellavia…
    Citation: Journal of Translational Medicine 2025 23:538
  19. Protein Tyrosine Phosphatase Receptor Type Kappa (PTPRK) is a membrane-bound tyrosine phosphatase encoded by the frequently deleted region of chromosome 6q, which plays a crucial role in regulating cell signaling...

    Authors: Chen Zheng, Ting Liu, An Qi Wang, Xing An Chen, Rong Zhe Zhang, Xuan Chao Wang, Chao Yue Lv, Ru Lu Pan, Ou Chen Wang and Xin-Cheng Lu
    Citation: Journal of Translational Medicine 2025 23:534
  20. The delivery of macromolecules through dermal and transdermal routes presents both significant challenges and transformative opportunities in therapeutic applications. This review highlights the most recent ad...

    Authors: Medha Bhat, Abhay Tharmatt, Samarth Bhargava, Tushar Kumeria, Amit Mishra, Anupama Mittal and Deepak Chitkara
    Citation: Journal of Translational Medicine 2025 23:513
  21. Tumor immunotherapy is one of the most promising strategies in cancer treatment. Specifically, intraperitoneal immunotherapy has emerged as a novel approach for dealing with abdominal metastases. Previously, w...

    Authors: Qi Wang, Yi Mei, Wenmei Rao, Sen Hong, Aoxing Chen, Yang Yang and Qin Liu
    Citation: Journal of Translational Medicine 2025 23:509
  22. Peptide-based therapeutics for melanoma have received increasing attention in medical research. However, the local delivery of such therapeutics poses unique challenges. Cell-penetrating peptides (CPPs) with t...

    Authors: Zheng Zhao, Zi-Qian Li, Ying-Bin Huang, Meng-meng Liu, Fei Cao, Guo-Long Bu, Peng-Fei Xu, Qi Fang, Zhu-Long Hu, Di Wu, Guo-Kai Feng and Xue-Kui Liu
    Citation: Journal of Translational Medicine 2025 23:495
  23. Despite the prevalence of non-small cell lung cancer (NSCLC) is high, the limited early detection and management of these tumors are restricted since there is an absence of reliable and precise diagnostic biom...

    Authors: Hongyuan Yin, Jiayi Shi, Shaoling Li, Qianhui You, Huici Zhu, Chinying Koo, Baonian Liu, Likun Hou and Chunyan Wu
    Citation: Journal of Translational Medicine 2025 23:490
  24. Dual-specific phosphatase-8 (DUSP8), identified as the first gene in a genome-wide association study (GWAS), is implicated in cellular oxidative stress, proliferation, apoptosis, and drug resistance through it...

    Authors: Tingping Cao, Quanling Zhou, Fujun Li, Mingyue Wang, Ming Zhang, Xiaohui Li, Hailong Zhao and Ya Zhou
    Citation: Journal of Translational Medicine 2025 23:485
  25. Melanoma is a highly aggressive form of skin cancer. Despite significant advances in targeted therapies and immunotherapeutic approaches, some patients still have poor response rates, making a deeper understan...

    Authors: Yongyi Xie, Ruoqi Wang, Mingyuan Xu, Jiashe Chen, Wei Tan, Yanbin Chen, Yun Bai, Nanhui Wu, Fei Wu, Xiaoxiang Xu, Xin Ma and Yeqiang Liu
    Citation: Journal of Translational Medicine 2025 23:470
  26. The ribosomal DNA (rDNA) plays a vital role in regulating protein synthesis by ribosome biogenesis, essential for maintaining cellular growth, metabolism, and more. Cancer cells show a high dependence on ribos...

    Authors: Hong-Xia Li, Yi-Meng He, Jing Fei, Man Guo, Chen Zeng, Pi-Jun Yan, Yong Xu, Gang Qin and Fang-Yuan Teng
    Citation: Journal of Translational Medicine 2025 23:457
  27. Endometrial cancer ranks among the most common gynecological cancers, with increasing rates of incidence and death. Cold atmospheric plasma (CAP) has become a promising novel therapeutic approach for cancer tr...

    Authors: Jinlin Liu, Yang Li, Ruonan Ma, Youming Chen, Jinyang Wang, Lindong Zhang, Baojin Wang, Zidi Zhang, Lili Huang, Hongyan Zhang, Junhu Wan and Hongyang Liu
    Citation: Journal of Translational Medicine 2025 23:442
  28. Breast cancer is one of the most prevalent malignant tumors among women and ranks as the second leading cause of cancer-related deaths in females, primarily due to delays in diagnosis and shortcomings in treat...

    Authors: Yun Hong, Jun He, Dan Deng, Qinyue Liu, Xuyu Zu and Yingying Shen
    Citation: Journal of Translational Medicine 2025 23:439
  29. Extracellular vesicles (EVs) produced in the tumor microenvironment in response to chemotherapy promote chemotherapy-resistant phenotypes. However, the role of EVs proteins induced by gastric cancer (GC) cell ...

    Authors: Yuhan Liao, Xinhua Chen, Hao Xu, Yunfei Zhi, Xinghua Zhuo, Jiang Yu and Liang Zhao
    Citation: Journal of Translational Medicine 2025 23:320
  30. Endothelial dysfunction is a hallmark feature of cardiovascular disease (CVD), yet the underlying mechanisms are still poorly understood. This has impeded the development of effective therapies, particularly f...

    Authors: Sahar Ghorbanpour, Siân Peta Cartland, Hao Chen, Sanchit Seth, Rupert C. Ecker, Claire Richards, Dunja Aksentijevic, Matthew P. Padula, Louise Cole, Majid Ebrahimi Warkiani, Mary Meltem Kavurma and Lana McClements
    Citation: Journal of Translational Medicine 2025 23:309
  31. Emerging evidence has confirmed the inextricable connection between N4-acetylcytidine (ac4C) mRNA modification and the clinical characteristics of malignancies. Nonetheless, it is uncertain whether and how ac4...

    Authors: Jianlan Liu, Pengpeng Zhang, Chaoqin Wu, Binlin Luo, Xiaojian Cao and Jian Tang
    Citation: Journal of Translational Medicine 2025 23:287
  32. Neisseria gonorrhoeae is an escalating global health threat due to increasing antimicrobial resistance. The emergence of multidrug-resistant (MDR) strains necessitates alternative prevention strategies. This stud...

    Authors: Narjes Noori Goodarzi, Seyed Mahmoud Barzi, Soheila Ajdary, Mohsen Chiani, Mir Saeed Yekaninejad, Farzad Badmasti and Mohammad Reza Pourmand
    Citation: Journal of Translational Medicine 2025 23:261
  33. Pentamidine (PTM) is an aromatic diamidine administered for infectious diseases, e.g. sleeping sickness, malaria, and Pneumocystis jirovecii pneumonia. Due to similarities of cellular mechanisms between human cel...

    Authors: Nima Rastegar-Pouyani, Mohammad Amin Farzin, Jaber Zafari, Mohadeseh Haji Abdolvahab and Shokoufeh Hassani
    Citation: Journal of Translational Medicine 2025 23:258
  34. Progression to castration resistance is the leading cause of death in prostate cancer patients. Long non-coding RNAs (lncRNAs) have recently become a focal point in the regulation of cancer development. Howeve...

    Authors: Yingying Wu, Shaojie Cheng, Ting Zhang, Leilei Wang, Ting Li, Yongbo Zheng, Guo Yang, Xiaohou Wu, Chunli Luo, Tingmei Chen and Liping Ou
    Citation: Journal of Translational Medicine 2025 23:255
  35. Transmembrane protein 52B (TMEM52B) is a novel gene expressed widely in various normal human tissues; however, the biological function of TMEM52B in cancer remains largely unknown. Previously, we demonstrated ...

    Authors: Yunhee Lee, Dongjoon Ko, Junghwa Yoon and Semi Kim
    Citation: Journal of Translational Medicine 2025 23:146
  36. CircRNAs are closely related to ferroptosis in gastric cancer cells; however, the mechanism by which circRNAs regulate ferroptosis in gastric carcinogenesis remains unknown. CircRNA-encoded novel peptides are ...

    Authors: Song Wang, Chengwei Wu, Jiawei Wang, Feng Yuan, Yinfen Hou, Tingting Cao, Lishuai Xu, Long Qian, Yabin Xia, Li Xu, Ailiang Zeng, Xiaoming Wang, Luman Wang and Xiaoxu Huang
    Citation: Journal of Translational Medicine 2025 23:250
  37. Co-stimulatory molecules are imperative for CD8+ T cells to eliminate target cell and maintain sustained cytotoxicity. Despite an advanced understanding of the co-stimulatory molecules deficiency that results in ...

    Authors: Hanyi Zeng, Jiaping Yu, Haijian Wang, Mengying Shen, Xuejing Zou, Ziyong Zhang and Li Liu
    Citation: Journal of Translational Medicine 2025 23:225
  38. Immune checkpoint inhibitors (ICIs) are emerging promising agents for the treatment of patients with esophageal squamous cell carcinoma (ESCC), however, there are only a small proportion respond to ICI therapy...

    Authors: Xinxin Cheng, Huihui Zhao, Zhangwang Li, Liping Yan, Qingjie Min, Qingnan Wu and Qimin Zhan
    Citation: Journal of Translational Medicine 2025 23:197
  39. Tyroservatide (YSV), a bioactive tripeptide, holds potential as an anti-tumor agent. However, its specific effects on oral squamous cell carcinoma (OSCC) have not been elucidated. This study aims to investigat...

    Authors: Lin Qiu, Qian Gao, Yiheng Liao, Xinxin Li and Cuiying Li
    Citation: Journal of Translational Medicine 2025 23:145
  40. Acute pancreatitis (AP) presents a significant clinical challenge with limited therapeutic options. The complex etiology and pathophysiology of AP emphasize the need for innovative treatments. This study explo...

    Authors: Amaya Lopez-Pascual, Eva Santamaria, Nuria Ardaiz, Iker Uriarte, Tiffany Palmer, Anne-Renee Graham, Celia Gomar, Roberto C. Barbero, M. Ujue Latasa, Maria Arechederra, Jesus M. Urman, Carmen Berasain, Antonio Fontanellas, Carlos L. del Rio, Maite G. Fernandez-Barrena, Paolo G.V. Martini…
    Citation: Journal of Translational Medicine 2025 23:122
  41. The high mortality rate of metastatic colorectal cancer (CRC) is primarily attributed to resistance to chemotherapy, where cancer stem cells (CSCs) play a crucial role. Deubiquitinating enzymes are essential r...

    Authors: Fengtian Li, Yi Zhou, Xinyue Lin, Yaxin Zhang, Qingyong Hu, Enen Zhao, Huali Li, Xingyan Pan, Feng Shu, Kun Zhang, Chengmei Huang, Na Tang and Wenting Liao
    Citation: Journal of Translational Medicine 2025 23:114
  42. The sustained activation of androgen receptor splice variant-7 (AR-V7) is a key factor in the resistance of castration-resistant prostate cancer (CRPC) to second-generation anti-androgens such as enzalutamide ...

    Authors: Xiannan Meng, Qingxuan Wu, Chengsong Cao, Wendong Yang, Sufang Chu, Hongjun Guo, Suhua Qi and Jin Bai
    Citation: Journal of Translational Medicine 2025 23:108
  43. Super enhancers are a unique class of enhancers that possess a distinct structure and mechanism, which enable them to exhibit stronger gene transcription regulatory function than classical enhancers, thereby r...

    Authors: Yumeng Ji, Baixue Li, Rongjin Lin, Jing Yuan, Yang Han, Yuping Du and Yang Zhao
    Citation: Journal of Translational Medicine 2025 23:98
  44. PRDX2 is significantly expressed in various cancers and is associated with the proliferation of tumor cells. Nonetheless, the precise mechanism of PRDX2 in tumor immunity remains incompletely understood. This ...

    Authors: Yunjia Dong, Anqi Cheng, Jiawei Zhou, Jianqiang Guo, Yafeng Liu, Xuan Li, Maoqian Chen, Dong Hu and Jing Wu
    Citation: Journal of Translational Medicine 2025 23:81
  45. This review seeks to elucidate the therapeutic potential of tumor necrosis factor receptor 1 (TNFR1) and enhance our comprehension of its role in disease mechanisms. As a critical cell-surface receptor, TNFR1 ...

    Authors: Yingying Li, Ruiwei Ye, Haorui Dai, Jiayi Lin, Yue Cheng, Yonghong Zhou and Yiming Lu
    Citation: Journal of Translational Medicine 2025 23:71
  46. Agonistic monoclonal antibodies targeting 4-1BB/CD137 have shown preclinical promise, but their clinical development has been limited by severe liver toxicity or limited efficacy. Therefore, a safe and efficie...

    Authors: Da-yan Zhang, Zhi-hua Zhang, Wen-ting Liu, Wei-ming Zhou, Peng-fei Zhou, Juan-juan Wei, Xue-jing Dai, Xiao-li Zeng, Yu-qiong Zhou, Han-wang Li, Heng Zhang, Ao-lin Shen, Lian-sheng Cheng, Guo-dong Shen and Yi-fu He
    Citation: Journal of Translational Medicine 2025 23:53
  47. Tumor-associated macrophages (TAMs) are pivotal immune cells within the tumor microenvironment (TME), exhibiting dual roles across various cancer types. Depending on the context, TAMs can either suppress tumor...

    Authors: Kaiting Chen, Xiaochen Li, Shuyi Dong, Yu Guo, Ziyin Luo, Shi-Min Zhuang, Jie Liu, Tianrun Liu, Jing Liao and Weiping Wen
    Citation: Journal of Translational Medicine 2025 23:27
  48. Glioblastoma (GBM) is a rare brain cancer with an exceptionally high mortality rate, which illustrates the pressing demand for more effective therapeutic options. Despite considerable research efforts on GBM, ...

    Authors: Shixue Sun, Zeenat Shyr, Kathleen McDaniel, Yuhong Fang, Dingyin Tao, Catherine Z. Chen, Wei Zheng and Qian Zhu
    Citation: Journal of Translational Medicine 2025 23:25