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Cell, Tissue and Gene Therapy

Section edited by David Stroncek

This section aims to improve communication between basic and clinical research related to cell, tissue and gene therapies. Effective clinical therapies require efficient translation of research between the bench and bedside to allow the discovery, development, scale up, and production of cells, tissues and vectors. These therapies are being used to treat patients with a range of diseases including cancer, cardiovascular diseases, autoimmune disorders.

Studies considered for publication include those regarding the nature of cells, tissues and vectors used in clinical therapies; methods to collect, isolate and modify cells; and novel reagents, equipment, and instruments used in production. The section also welcomes research into the application of clinical therapies; factors that effect the safety and efficacy; in particular the potency, stability and purity of cells or tissue; and issues related to the safety of cell and tissue donors.

Featured articles

Phase I and registry study of autologous bone marrow concentrate evaluated in PDE5 inhibitor refractory erectile dysfunction

Research | Mark Bieri et al


Rationale for the clinical use of adipose-derived mesenchymal stem cells for COVID-19 patients​​​​​​​

Review | Christopher J. Rogers et al


Allo-priming as a universal anti-viral vaccine: protecting elderly from current COVID-19 and any future unknown viral outbreak

Review |  Michael Har-Noy & Reuven Or 

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  1. Abnormal gene expression level or expression of genes containing deleterious mutations are two of the main determinants which lead to genetic disease. To obtain a therapeutic effect and thus to cure genetic di...

    Authors: Hsing-Chuan Tsai, Violena Pietrobon, Maoyu Peng, Suning Wang, Lihong Zhao, Francesco M. Marincola and Qi Cai
    Citation: Journal of Translational Medicine 2022 20:535
  2. Circular ribonucleic acids (circRNAs) are a class of long non-coding RNA that were once regarded as non-functional transcription byproducts. However, recent studies suggested that circRNAs may exhibit importan...

    Authors: Ryan Wong, Yiwen Zhang, Hailin Zhao and Daqing Ma
    Citation: Journal of Translational Medicine 2022 20:533
  3. Chronic graft-versus-host disease (GVHD) is a major cause of late morbidity and non-relapse mortality in recipients of allogeneic hematopoietic cell transplantation (HCT). Its biology, however, remains poorly ...

    Authors: Alain Mina, Lauren Curtis, Kamille West, Yu Ying Yau, Edward W. Cowen, Frances Hakim and Steven Z. Pavletic
    Citation: Journal of Translational Medicine 2022 20:519
  4. Osteoarthritis (OA) is a chronic degenerative joint disease characterized by the destruction of the articular cartilage, sclerosis of the subchondral bone, and joint dysfunction. Its pathogenesis is attributed...

    Authors: Jianghong Huang, Qisong Liu, Jiang Xia, Xi Chen, Jianyi Xiong, Lei Yang and Yujie Liang
    Citation: Journal of Translational Medicine 2022 20:515
  5. Clinical CAR T-cell therapy using integrating vector systems represents a promising approach for the treatment of hematological malignancies. Lentiviral and γ-retroviral vectors are the most commonly used vect...

    Authors: Lipei Shao, Rongye Shi, Yingdong Zhao, Hui Liu, Alexander Lu, Jinxia Ma, Yihua Cai, Tatyana Fuksenko, Alejandra Pelayo, Nirali N. Shah, James N. Kochenderfer, Scott M. Norberg, Christian Hinrichs, Steven L. Highfill, Robert P Somerville, Sandhya R. Panch…
    Citation: Journal of Translational Medicine 2022 20:514
  6. Immune checkpoint inhibitors are promising tools in combating several cancers, including head and neck squamous cell carcinoma (HNSCC). However, a substantial portion of HNSCC patients do not respond to PD-L1 ...

    Authors: Shuang Li, Ding Wang, Jinzhang Cheng, Jicheng Sun, Dhan V. Kalvakolanu, Xue Zhao, Di Wang, Yunhan You, Ling Zhang and Dan Yu
    Citation: Journal of Translational Medicine 2022 20:505
  7. Autologous hematopoietic progenitor cell (HPC) transplantation is currently the standard of care for a fraction of patients with newly diagnosed myelomas and relapsed or refractory lymphomas. After high-dose c...

    Authors: Bechara Mfarrej, Olivier Vicari, Sarah Ouffai, Carine Malenfant, Angela Granata, Sophie Thevenet, Christian Chabannon, Claude Lemarié and Boris Calmels
    Citation: Journal of Translational Medicine 2022 20:503
  8. Chimeric antigen receptor (CAR)-T cell therapy is a powerful adoptive immunotherapy against both B-cell malignancies and some types of solid tumors. Interleukin (IL) -15 is an important immune stimulator that ...

    Authors: Ying Zhang, Qinghui Zhuang, Fang Wang, Can Zhang, Chang Xu, Aiqin Gu, William H. Zhong, Yi Hu and Xiaosong Zhong
    Citation: Journal of Translational Medicine 2022 20:432
  9. Currently, there is no regenerative therapy for patients with neurological and neurodegenerative disorders. Cell-therapies have emerged as a potential treatment for numerous brain diseases. Despite recent adva...

    Authors: Ruslan Rust, Rebecca Z. Weber, Melanie Generali, Debora Kehl, Chantal Bodenmann, Daniela Uhr, Debora Wanner, Kathrin J. Zürcher, Hirohide Saito, Simon P. Hoerstrup, Roger M. Nitsch and Christian Tackenberg
    Citation: Journal of Translational Medicine 2022 20:421
  10. Increasing evidence supports the concept of prenatal programming as an early factor in the aging process. DNA methylation age (DNAm age), global genome-wide DNA methylation (global methylation), telomere lengt...

    Authors: Brigitte Reimann, Dries S. Martens, Congrong Wang, Akram Ghantous, Zdenko Herceg, Michelle Plusquin and Tim S. Nawrot
    Citation: Journal of Translational Medicine 2022 20:353
  11. Cytokine release syndrome (CRS) is a strong immune system response that can occur as a result of the reaction of a cellular immunotherapy with malignant cells. While the frequency and management of CRS in CAR ...

    Authors: Lipei Shao, Alejandra Pelayo, Rongye Shi, Jinxia Ma, Hui Liu, Yihua Cai, Michaela Prochazkova, Robert P. Somerville, Sandhya R. Panch, Nirali N. Shah, David F. Stroncek and Ping Jin
    Citation: Journal of Translational Medicine 2022 20:338
  12. Despite promising results in clinical studies, the mechanism for the beneficial effects of allogenic cell-based therapies remains unclear. Macrophages are not only critical mediators of inflammation but also c...

    Authors: Rachana Mishra, Progyaparamita Saha, Srinivasa Raju Datla, Pranav Mellacheruvu, Muthukumar Gunasekaran, Sameer Ahmad Guru, Xubin Fu, Ling Chen, Roberto Bolli, Sudhish Sharma and Sunjay Kaushal
    Citation: Journal of Translational Medicine 2022 20:323
  13. Radiation-induced skin injury is a well-known risk factor for impaired wound healing. Over time, the deleterious effects of radiation on skin produce a fibrotic, hypovascular dermis poorly suited to wound heal...

    Authors: Hendrik Lintel, Darren B. Abbas, Christopher V. Lavin, Michelle Griffin, Jason L. Guo, Nicholas Guardino, Andrew Churukian, Geoffrey C. Gurtner, Arash Momeni, Michael T. Longaker and Derrick C. Wan
    Citation: Journal of Translational Medicine 2022 20:274
  14. Tumor-draining lymph nodes (TDLNs) are primary sites, where anti-tumor lymphocytes are primed to tumor-specific antigens and play pivotal roles in immune responses against tumors. Although adoptive cell therap...

    Authors: Kazumi Okamura, Satoshi Nagayama, Tomohiro Tate, Hiu Ting Chan, Kazuma Kiyotani and Yusuke Nakamura
    Citation: Journal of Translational Medicine 2022 20:241
  15. As a promising alternative platform for cellular immunotherapy, natural killer cells (NK) have recently gained attention as an important type of innate immune regulatory cell. NK cells can rapidly kill multipl...

    Authors: Junfeng Chu, Fengcai Gao, Meimei Yan, Shuang Zhao, Zheng Yan, Bian Shi and Yanyan Liu
    Citation: Journal of Translational Medicine 2022 20:240
  16. Chimeric antigen receptor (CAR) T cell therapy has attracted attention for its promising therapeutic effects on hematological malignancies. However, there are problems such as relapse during long-term follow-u...

    Authors: Taku Kouro, Hidetomo Himuro and Tetsuro Sasada
    Citation: Journal of Translational Medicine 2022 20:239
  17. The two oncogenic human gammaherpesviruses, Kaposi sarcoma-associated herpesvirus (KSHV) and Epstein–Barr virus (EBV), both downregulate immune surface molecules, such as MHC-I, ICAM-1, and B7-2, enabling them...

    Authors: Yiquan Wu, Prabha Shrestha, Natalie M. Heape and Robert Yarchoan
    Citation: Journal of Translational Medicine 2022 20:217
  18. Cellular therapies have become an important part of clinical care. The treatment of patients with cell therapies often involves the collection of autologous cells at the medical center treating the patient, th...

    Authors: David Stroncek, Anh Dinh, Herleen Rai, Nan Zhang, Rob Somerville and Sandhya Panch
    Citation: Journal of Translational Medicine 2022 20:184
  19. Diabetes mellitus (DM), currently affecting 463 million people worldwide is a chronic disease characterized by impaired glucose metabolism resulting from the loss or dysfunction of pancreatic β-cells with the ...

    Authors: Abdoulaye Diane, Noora Ali Al-Shukri, Razik Bin Abdul Mu-u-min and Heba H. Al-Siddiqi
    Citation: Journal of Translational Medicine 2022 20:163
  20. The NK cell line NK-92 and its genetically modified variants are receiving attention as immunotherapies to treat a range of malignancies. However, since NK-92 cells are themselves tumors, they require irradiat...

    Authors: Lydia Navarrete-Galvan, Michael Guglielmo, Judith Cruz Amaya, Julie Smith-Gagen, Vincent C. Lombardi, Rebecca Merica and Dorothy Hudig
    Citation: Journal of Translational Medicine 2022 20:151
  21. Nanocarriers as drug/biomolecule delivery systems have been significantly developed during recent decades. Given the stability, reasonable delivery efficiency, and safety of nanocarriers, there are several bar...

    Authors: Ala Amiri, Rafieh Bagherifar, Ehsan Ansari Dezfouli, Seyed Hossein Kiaie, Reza Jafari and Reihaneh Ramezani
    Citation: Journal of Translational Medicine 2022 20:125
  22. Chimeric antigen receptor (CAR) T-cell therapy has proven to be a valuable new treatment option for patients with B-cell malignancies. However, by applying selective pressure, outgrowth of antigen-negative tum...

    Authors: Gils Roex, Diana Campillo-Davo, Donovan Flumens, Philip Anthony Gilbert Shaw, Laurens Krekelbergh, Hans De Reu, Zwi N. Berneman, Eva Lion and Sébastien Anguille
    Citation: Journal of Translational Medicine 2022 20:124
  23. The transformation of hepatic stellate cell (HSC) to myofibroblast is a key event during liver fibrogenesis. However, the differentiation trajectory of HSC-to-myofibroblast transition and the switching genes d...

    Authors: Hua Wang, Shaoping Zheng, Hongbo Jiang, Xuejia Wang, Fengqin Zhou and Zhihong Weng
    Citation: Journal of Translational Medicine 2022 20:53
  24. The use of cellular therapies to treat cancer, inherited immune deficiencies, hemoglobinopathies and viral infections is growing rapidly. The increased interest in cellular therapies has led to the development...

    Authors: David F. Stroncek, Robert P. T. Somerville and Steven L. Highfill
    Citation: Journal of Translational Medicine 2022 20:34
  25. A growing number of clinical trials have shown that regulatory T (Treg) cell transfer may have a favorable effect on the maintenance of self-tolerance and immune homeostasis in different conditions such as graft-...

    Authors: Cristiana Lavazza, Silvia Budelli, Elisa Montelatici, Mariele Viganò, Francesca Ulbar, Lucia Catani, Marta Giulia Cannone, Sara Savelli, Elisa Groppelli, Lorenza Lazzari, Roberto M. Lemoli, Matteo Cescon, Gaetano La Manna, Rosaria Giordano and Tiziana Montemurro
    Citation: Journal of Translational Medicine 2022 20:14
  26. Chimeric antigen receptor (CAR) or T-cell receptor (TCR) engineered T-cell therapy has recently emerged as a promising adoptive immunotherapy approach for the treatment of hematologic malignancies and solid tu...

    Authors: Yihua Cai, Michaela Prochazkova, Chunjie Jiang, Hannah W. Song, Jianjian Jin, Larry Moses, Nikolaos Gkitsas, Robert P. Somerville, Steven L. Highfill, Sandhya Panch, David F. Stroncek and Ping Jin
    Citation: Journal of Translational Medicine 2021 19:523
  27. Knee osteoarthritis is the most prevalent joint disease and a frequent cause of pain, functional loss and disability. Conventional treatments have demonstrated only modest clinical benefits whereas cell-based ...

    Authors: José María Lamo-Espinosa, Felipe Prósper, Juan F. Blanco, Fermín Sánchez-Guijo, Mercedes Alberca, Verónica García, Margarita González-Vallinas and Javier García-Sancho
    Citation: Journal of Translational Medicine 2021 19:506
  28. Chimeric antigen receptor T cell (CAR-T cell) therapy is a relatively new, effective, and rapidly evolving therapeutic for adoptive immunotherapies. Although it has achieved remarkable effect in hematological ...

    Authors: Meng Zhang, Xin Jin, Rui Sun, Xia Xiong, Jiaxi Wang, Danni Xie and MingFeng Zhao
    Citation: Journal of Translational Medicine 2021 19:499
  29. CAR T-cell therapy has been recently unveiled as one of the most promising cancer therapies in hematological malignancies. However, solid tumors mount a profound line of defense to escape immunosurveillance by...

    Authors: Khadijeh Alishah, Matthias Birtel, Elham Masoumi, Leila Jafarzadeh, Hamid Reza Mirzaee, Jamshid Hadjati, Ralf-Holger Voss, Mustafa Diken and Sedighe Asad
    Citation: Journal of Translational Medicine 2021 19:482
  30. Gene transfer is an important tool for cellular therapies. Lentiviral vectors are most effectively transferred into lymphocytes or hematopoietic progenitor cells using spinoculation. To enable cGMP (current Go...

    Authors: Victoria Ann Remley, Jianjian Jin, Sarmila Sarkar, Larry Moses, Michaela Prochazkova, Yihua Cai, Lipei Shao, Hui Liu, Tatyana Fuksenko, Ping Jin, David F. Stroncek and Steven L. Highfill
    Citation: Journal of Translational Medicine 2021 19:474
  31. Nucleotide mutations in the ABO gene may reduce the activity of glycosyltransferase, resulting in lower levels of A or B antigen expression in red blood cells. Six known splice sites have been identified accordin...

    Authors: Xiaozhen Hong, Yanling Ying, Jingjing Zhang, Shu Chen, Xianguo Xu, Ji He and Faming Zhu
    Citation: Journal of Translational Medicine 2021 19:470
  32. Adoptive transfer of chimeric antigen receptor (CAR)-engineered T cells combined with checkpoint inhibition may prevent T cell exhaustion and improve clinical outcomes. However, the approach is limited by cumu...

    Authors: Zhifen Yang, Lingyu Li, Ahu Turkoz, Pohan Chen, Rona Harari-Steinfeld, Maggie Bobbin, Ofir Stefanson, Hana Choi, Violena Pietrobon, Bennett Alphson, Angshumala Goswami, Vitaly Balan, Alper Kearney, Dharmesh Patel, Jin Yang, Damla Inel…
    Citation: Journal of Translational Medicine 2021 19:459
  33. HIV-1 reservoirs persist in the presence of combined antiretroviral therapy (cART). However, cART has transformed HIV-1 infection into a chronic disease marked by control of HIV-1 viral load and mortality redu...

    Authors: Matthew Weichseldorfer, Yvonne Affram, Alonso Heredia, Zahra Rikhtegaran-Tehrani, Mohammad M. Sajadi, Sumiko P. Williams, Yutaka Tagaya, Francesca Benedetti, Habib O. Ramadhani, Frank Denaro, Arshi Munawwar, Joseph Bryant, Davide Zella, Marvin Reitz, Fabio Romerio and Olga S. Latinovic
    Citation: Journal of Translational Medicine 2021 19:453
  34. The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada...

    Authors: Jin-A Lee, Alex Cho, Elena N. Huang, Yiming Xu, Henry Quach, Jim Hu and Amy P. Wong
    Citation: Journal of Translational Medicine 2021 19:452
  35. Hypertrophic scar (HTS) is a fibrotic disorder of skins and may have repercussions on the appearance as well as functions of patients. Recent studies related have shown that competitive endogenous RNA (ceRNA) ...

    Authors: Zewei Zhang, Xin Huang, Jiahao Yang, Shuchen Gu, Yixuan Zhao, Yunhan Liu, Yimin Khoong, Shuqi Wang, Shenying Luo, Tao Zan and Guangshuai Li
    Citation: Journal of Translational Medicine 2021 19:451

    The Correction to this article has been published in Journal of Translational Medicine 2022 20:35

  36. Established clinical assessments for detecting dementia risk often require time, cost, and face-to-face meetings. We aimed to develop a Simplified Telephone Assessment for Dementia risk (STAD) (a new screening...

    Authors: Keitaro Makino, Sangyoon Lee, Seongryu Bae, Ippei Chiba, Kenji Harada, Osamu Katayama, Yohei Shinkai and Hiroyuki Shimada
    Citation: Journal of Translational Medicine 2021 19:448
  37. In the ovarian follicle, the Theca Cells (TCs) have two main functions: preserving morphological integrity and, importantly, secreting steroid androgen hormones. TCs express the essential enzyme 17α-hydroxylas...

    Authors: Christian Secchi, Martina Belli, Tracy N. H. Harrison, Joseph Swift, CheMyong Ko, Antoni J. Duleba, Dwayne Stupack, R. Jeffrey Chang and Shunichi Shimasaki
    Citation: Journal of Translational Medicine 2021 19:428
  38. The present study aimed to compare the efficacy and safety profiles of microspheres versus (vs.) polyvinyl alcohol (PVA) for bronchial artery embolization (BAE) treatment in patients with hemoptysis.

    Authors: Zhigang Fu, Xun Li, Fei Cai, Yinpeng Yuan, Xiaolin Zhang, Jingxia Qin and Yonghui Liang
    Citation: Journal of Translational Medicine 2021 19:422
  39. Systemic sclerosis is an autoimmune disease characterized by excessive dermal fibrosis with progression to internal organs, vascular impairment and immune dysregulation evidenced by the infiltration of inflamm...

    Authors: Pauline Beurier, Laure Ricard, Deborah Eshagh, Florent Malard, Lama Siblany, Olivier Fain, Mohamad Mohty, Béatrice Gaugler and Arsène Mekinian
    Citation: Journal of Translational Medicine 2021 19:375
  40. Recently, mesenchymal stem/stromal cells (MSCs) due to their pro-angiogenic, anti-apoptotic, and immunoregulatory competencies along with fewer ethical issues are presented as a rational strategy for regenerat...

    Authors: Soudeh Moghadasi, Marischa Elveny, Heshu Sulaiman Rahman, Wanich Suksatan, Abduladheem Turki Jalil, Walid Kamal Abdelbasset, Alexei Valerievich Yumashev, Siavash Shariatzadeh, Roza Motavalli, Farahnaz Behzad, Faroogh Marofi, Ali Hassanzadeh, Yashwant Pathak and Mostafa Jarahian
    Citation: Journal of Translational Medicine 2021 19:302
  41. Genetically engineered chimeric antigen receptor (CAR) T lymphocytes are promising therapeutic tools for cancer. Four CAR T cell drugs, including tisagenlecleucel (tisa-cel) and axicabtagene-ciloleucel (axi-ce...

    Authors: Rafik Haderbache, Walid Warda, Eric Hervouet, Mathieu Neto da Rocha, Rim Trad, Vincent Allain, Clementine Nicod, Catherine Thieblemeont, Nicolas Boissel, Pauline Varlet, Ibrahim Yakoub Agha, Lucie Bouquet, Melanie Guiot, Fabienne Venet, Pierre Sujobert, Xavier Roussel…
    Citation: Journal of Translational Medicine 2021 19:265
  42. Adipose cell-free derivatives have been recently gaining attention as potential therapeutic agents for various human diseases. In this context, mesenchymal stromal/stem cells (MSCs), adipocyte mesenchymal stem...

    Authors: Loubna Mazini, Luc Rochette and Gabriel Malka
    Citation: Journal of Translational Medicine 2021 19:234
  43. Transfusion-related immunomodulation (TRIM) can be caused by exosomes, in which case, microRNAs (miRNAs) are one critical factor impacting exosome behavior. This study aims to investigate and analyze the expre...

    Authors: Yujie Kong, Xue Tian, Rui He, Chenyue Li, Haixia Xu, Li Tian and Zhong Liu
    Citation: Journal of Translational Medicine 2021 19:225
  44. Mitochondria are essential organelles that provide energy for cellular functions, participate in cellular signaling and growth, and facilitate cell death. Based on their multifactorial roles, mitochondria are ...

    Authors: Kei Hayashida, Ryosuke Takegawa, Muhammad Shoaib, Tomoaki Aoki, Rishabh C. Choudhary, Cyrus E. Kuschner, Mitsuaki Nishikimi, Santiago J. Miyara, Daniel M. Rolston, Sara Guevara, Junhwan Kim, Koichiro Shinozaki, Ernesto P. Molmenti and Lance B. Becker
    Citation: Journal of Translational Medicine 2021 19:214
  45. Due to mTOR (mammalian/mechanistic target of rapamycin) gene-loss mice die during embryonic development, the role of mTOR in platelets has not been evaluated using gene knockout technology.

    Authors: Longsheng Wang, Gang Liu, Nannan Wu, Baiyun Dai, Shuang Han, Qiaoyun Liu, Fang Huang, Zhihua Chen, Weihong Xu, Dajing Xia and Cunji Gao
    Citation: Journal of Translational Medicine 2021 19:201
  46. The mortality rate of critically ill patients with acute respiratory distress syndrome (ARDS) is 30.9% to 46.1%. The emergence of the coronavirus disease 2019 (Covid-19) has become a global issue with raising ...

    Authors: Wendi Wang, Wei Lei, Lina Jiang, Siqi Gao, Shijun Hu, Zi-Gang Zhao, Chun-Yu Niu and Zhen-Ao Zhao
    Citation: Journal of Translational Medicine 2021 19:198
  47. Endogenous Peptide Inhibitor of CXCR4 (EPI-X4) is a natural antagonist of the CXC chemokine receptor 4 (CXCR4). EPI-X4 is a 16-mer peptide that is released from human serum albumin (HSA) by acidic aspartic pro...

    Authors: Andrea Gilg, Mirja Harms, Lia-Raluca Olari, Ann-Kathrin Urbanowitz, Halvard Bonig and Jan Münch
    Citation: Journal of Translational Medicine 2021 19:190

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