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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. 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
    Content type: Review Published on:

  2. 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
    Content type: Research Published on:

  3. 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
    Content type: Research Published on:

  4. 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
    Content type: Commentary Published on:

  5. 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
    Content type: Methodology Published on:

  6. 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
    Content type: Research Published on:

  7. 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
    Content type: Commentary Published on:

  8. 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
    Content type: Review Published on:

  9. 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
    Content type: Research Published on:

  10. 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
    Content type: Research Published on:

  11. 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
    Content type: Research Published on:

  12. 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
    Content type: Research Published on:

  13. 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
    Content type: Research Published on:

  14. 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
    Content type: Review Published on:

  15. 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
    Content type: Research Published on:

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

  16. 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
    Content type: Research Published on:

  17. 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
    Content type: Research Published on:

  19. 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
    Content type: Research Published on:

  20. 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
    Content type: Research Published on:

  21. 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
    Content type: Review Published on:

  22. 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
    Content type: Methodology Published on:

  23. 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
    Content type: Commentary Published on:

  24. 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
    Content type: Research Published on:

  25. 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
    Content type: Review Published on:

  26. 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
    Content type: Research Published on:

  27. 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
    Content type: Review Published on:

  29. 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
    Content type: Research Published on:

  30. Cetuximab has been approved for use for first-line treatment of patients with wild-type KRAS metastatic colorectal cancer (CRC). However, treatment with cetuximab has shown limited efficacy as a CRC monotherapy. ...

    Authors: Shiqi Long, Yangzhuo Gu, Yuanyuan An, Xiaojin Lin, Xiaoqing Chen, Xianyao Wang, Chunxiang Liao, Weiwei Ouyang, Nianxue Wang, Zhixu He and Xing Zhao
    Citation: Journal of Translational Medicine 2021 19:185
    Content type: Research Published on:

  31. Spinal cord injury (SCI) is a severe traumatic disease of the central nervous system, with a global prevalence of 236–4187 per million people. This meta-analysis aimed to evaluate the safety and efficacy of me...

    Authors: Wei-can Chen, Wei-feng Liu, Yu-yan Bai, Ying-ying Zhou, Yan Zhang, Cong-mei Wang, Shu Lin and He-fan He
    Citation: Journal of Translational Medicine 2021 19:178
    Content type: Review Published on:

  32. The onset of the SARS-CoV-2 pandemic has resulted in ever-increasing casualties worldwide, and after 15 months, standard therapeutic regimens are yet to be discovered.

    Authors: Monire Jamalkhah, Yasaman Asaadi, Mohammadreza Azangou-Khyavy, Javad Khanali, Masoud Soleimani, Jafar Kiani and Ehsan Arefian
    Citation: Journal of Translational Medicine 2021 19:164
    Content type: Review Published on:

  33. Multipotent mesenchymal stromal cells (MSCs) are promising cellular therapeutics for the treatment of inflammatory and degenerative disorders due to their anti-inflammatory, immunomodulatory and regenerative p...

    Authors: Nerea Cuesta-Gomez, Gerard J. Graham and John D. M. Campbell
    Citation: Journal of Translational Medicine 2021 19:156
    Content type: Review Published on:

  34. Mesenchymal stem cells derived from human umbilical cord (hUC-MSCs) have immunomodulatory properties that are of interest to treat novel coronavirus disease 2019 (COVID-19). Leng et al. recently reported that ...

    Authors: Jonathan J. Hernandez, Doyle E. Beaty, Logan L. Fruhwirth, Ana P. Lopes Chaves and Neil H. Riordan
    Citation: Journal of Translational Medicine 2021 19:149
    Content type: Research Published on:

  35. Because of limitations of transportation imposed by the COVID-19 pandemic, current recommendation calls for cryopreservation of allogeneic stem cell transplants before patient conditioning. A single cell thera...

    Authors: Eliza Wiercinska, Vera Schlipfenbacher, Gesine Bug, Peter Bader, Mareike Verbeek, Erhard Seifried and Halvard Bonig
    Citation: Journal of Translational Medicine 2021 19:145
    Content type: Research Published on:

  36. Glioma is a complex cancer with a high morbidity and high mortality. Bone marrow mesenchymal stem cells (BMSCs) have shown promise as an excellent cell/drug delivery vehicle for gene-targeted therapy; however,...

    Authors: Jianjun Wu, Shoupin Xie, Hailong Li, Yanxia Zhang, Jia Yue, Chunlu Yan, Kai Liu, Yongqi Liu, Rui Xu and Guisen Zheng
    Citation: Journal of Translational Medicine 2021 19:143
    Content type: Research Published on:

  38. Although chimeric antigen receptor (CAR)-T cell therapy has been remarkably successful for haematological malignancies, its efficacy against solid tumors is limited. The combination of CAR-T cell therapy with ...

    Authors: Yajun Zhang, Pei Wang, Tengjiao Wang, Yuan Fang, Yongmei Ding and Qijun Qian
    Citation: Journal of Translational Medicine 2021 19:82
    Content type: Research Published on:

  39. Skeletal muscle aging is associated with a decline in motor function and loss of muscle mass- a condition known as sarcopenia. The underlying mechanisms that drive this pathology are associated with a failure ...

    Authors: Colin Harper, Venkatesh Gopalan and Jorming Goh
    Citation: Journal of Translational Medicine 2021 19:71
    Content type: Review Published on:

  40. Cutaneous wounds in patients with diabetes exhibit impaired healing due to physiological impediments and conventional care options are severely limited. Multipotent stromal cells (MSCs) have been touted as a p...

    Authors: Bibi S. Subhan, Jennifer Kwong, Joseph F. Kuhn, Arie Monas, Sonali Sharma and Piul S. Rabbani
    Citation: Journal of Translational Medicine 2021 19:16
    Content type: Research Published on:

  41. Currently, there are limited reports regarding investigation of the biological properties of polyetheretherketone (PEEK) coated with titanium (Ti) and hydroxyapatite (HA) in human. The objective of this study ...

    Authors: Ce Zhu, Miaomiao He, Lili Mao, Tao Li, Li Zhang, Limin Liu, Ganjun Feng and Yueming Song
    Citation: Journal of Translational Medicine 2021 19:14
    Content type: Research Published on:

  42. Healthy volunteer registry donors have become the backbone of stem cell transplantation programs. While most registrants will never become actual donors, a small minority are called upon twice, most commonly f...

    Authors: Soo-Zin Kim-Wanner, Seo-Youn Lee, Erhard Seifried and Halvard Bonig
    Citation: Journal of Translational Medicine 2020 18:487
    Content type: Research Published on:

  43. Myeloid-derived suppressor cells (MDSCs) play a critical role in modulating the immune response and promoting immune tolerance in models of autoimmunity and transplantation. Regulatory T cells (Tregs) exert th...

    Authors: Min-Jung Park, Jin-Ah Baek, Se-Young Kim, Kyung-Ah Jung, Jeong Won Choi, Sung-Hwan Park, Seung‐Ki Kwok and Mi-La Cho
    Citation: Journal of Translational Medicine 2020 18:483
    Content type: Research Published on:

  44. Periodontitis, a bacterium-induced inflammatory disease that is characterized by alveolar bone loss, is highly prevalent worldwide. Elucidating the underlying mechanisms of alveolar bone loss in periodontitis ...

    Authors: Xiaofei Huang, Mengru Xie, Yanling Xie, Feng Mei, Xiaofeng Lu, Xiaoshuang Li and Lili Chen
    Citation: Journal of Translational Medicine 2020 18:479
    Content type: Review Published on:

  45. During the coronavirus disease-2019 (COVID-19) pandemic, Italian hospitals faced the most daunting challenges of their recent history, and only essential therapeutic interventions were feasible. From March to ...

    Authors: Giuseppe Astori, Martina Bernardi, Angela Bozza, Daniela Catanzaro, Katia Chieregato, Anna Merlo, Monica Santimaria, Roberto Barbazza, Giuseppe Amodeo, Rachele Ciccocioppo, Francesca Elice and Marco Ruggeri
    Citation: Journal of Translational Medicine 2020 18:451
    Content type: Research Published on:

  46. Mesenchymal stem cells (MSCs) have captured great attention in regenerative medicine for over a few decades by virtue of their differentiation capacity, potent immunomodulatory properties, and their ability to...

    Authors: Sepideh Nikfarjam, Jafar Rezaie, Naime Majidi Zolbanin and Reza Jafari
    Citation: Journal of Translational Medicine 2020 18:449
    Content type: Review Published on:

  47. Vectors derived from adeno-associated viruses (AAVs) are widely used for gene transfer both in vitro and in vivo and have gained increasing interest as shuttle systems to deliver therapeutic genes to the heart...

    Authors: Zihou Liu, Kristin Klose, Sebastian Neuber, Meng Jiang, Manfred Gossen and Christof Stamm
    Citation: Journal of Translational Medicine 2020 18:437
    Content type: Methodology Published on:

  48. Extracellular vesicles are heterogeneous populations of naturally occurring secreted small vesicles. EVs function as signaling platforms to facilitate intracellular communication, which indicates the physiolog...

    Authors: Kaiyuan Xu, Qin Liu, Kaihui Wu, Liu Liu, Maomao Zhao, Hui Yang, Xiang Wang and Wenmei Wang
    Citation: Journal of Translational Medicine 2020 18:432
    Content type: Review Published on:

  49. Glioblastoma multiforme (GBM) is the most common malignant brain cancer that invades normal brain tissue and impedes surgical eradication, resulting in early local recurrence and high mortality. In addition, m...

    Authors: Craig A. Land, Phillip R. Musich, Dalia Haydar, Giedre Krenciute and Qian Xie
    Citation: Journal of Translational Medicine 2020 18:428
    Content type: Review Published on:

  50. Cancer vaccines provide a complex source of neoantigens. Still, increasing evidence reveals that the neoantigen quality rather than the quantity is predictive for treatment outcome.

    Authors: Inken Salewski, Yvonne Saara Gladbach, Steffen Kuntoff, Nina Irmscher, Olga Hahn, Christian Junghanss and Claudia Maletzki
    Citation: Journal of Translational Medicine 2020 18:402
    Content type: Research Published on:
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