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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. Spinal cord injury (SCI) is one of the most severe central nervous system injuries. Currently, transplanting bone marrow mesenchymal stem cells (BMSCs) is considered a therapeutic option for SCI. Tanshinone II...

    Authors: Xue-Mei Zhang, Jiao Ma, Yang Sun, Bing-Qian Yu, Zhuo-Min Jiao, Duo Wang, Mei-Yu Yu, Jin-Yue Li and Jin Fu
    Citation: Journal of Translational Medicine 2018 16:193
  2. To explore the mechanism of the different clinical efficacies of mesenchymal stem cell transplantation (MSCT) and identify a possible serum biomarker for predicting the therapeutic effect of MSCT in rheumatoid...

    Authors: Yi Yang, Xiao He, Rongseng Zhao, Wei Guo, Ming Zhu, Wei Xing, Dongpo Jiang, Chongyang Liu and Xiang Xu
    Citation: Journal of Translational Medicine 2018 16:165

    The Correction to this article has been published in Journal of Translational Medicine 2021 19:402

  3. The reconstruction of bone defects is often impaired by radiotherapy since bone quality is compromised by radiation. This study aims to investigate the therapeutic efficacy of the composite cell sheets-bone ma...

    Authors: Huan Liu, Yang Jiao, Wei Zhou, Shizhu Bai, Zhihong Feng, Yan Dong, Qian Liu, Xiaoke Feng and Yimin Zhao
    Citation: Journal of Translational Medicine 2018 16:137
  4. Cell homing is the mechanism by which an injury releases signaling molecules that cause recruitment, proliferation, migration and differentiation of progenitor cells. Stromal derived factor-1 (SDF-1) and its r...

    Authors: Melissa Kristocheck, Lucinara D. Dias, Carine Ghem, Bruna Eibel, Renato A. K. Kalil and Melissa M. Markoski
    Citation: Journal of Translational Medicine 2018 16:133
  5. Stem cell therapy has been applied in the treatment of acute-on-chronic liver failure (ACLF). However, its clinical efficiency is still debatable. The aim of this systematic review and meta-analysis is to eval...

    Authors: Ran Xue, Qinghua Meng, Jinling Dong, Juan Li, Qinwei Yao, Yueke Zhu and Hongwei Yu
    Citation: Journal of Translational Medicine 2018 16:126
  6. A major complication after allogeneic hematopoietic stem cell transplantation (aSCT) is the reactivation of herpesviruses such as cytomegalovirus (CMV) and Epstein–Barr virus (EBV). Both viruses cause signific...

    Authors: Regina Gary, Michael Aigner, Stephanie Moi, Stefanie Schaffer, Anja Gottmann, Stefanie Maas, Robert Zimmermann, Jürgen Zingsem, Julian Strobel, Andreas Mackensen, Josef Mautner, Andreas Moosmann and Armin Gerbitz
    Citation: Journal of Translational Medicine 2018 16:124
  7. The role of rabbit synovial fluid-derived mesenchymal stem cells (rbSF-MSCs) in cartilage defect repair remains undefined. This work evaluates the in vivo effects of rbSF-MSCs to repair knee articular cartilag...

    Authors: Zhaofeng Jia, Qisong Liu, Yujie Liang, Xingfu Li, Xiao Xu, Kan Ouyang, Jianyi Xiong, Daping Wang and Li Duan
    Citation: Journal of Translational Medicine 2018 16:123
  8. Pancreatic ductal adenocarcinoma (PDAC) has been increasing by 0.5% per year in the United States. PDAC portends a dismal prognosis and novel therapies are needed. This study describes the generation and chara...

    Authors: Michael P. O’Leary, Audrey H. Choi, Sang-In Kim, Shyambabu Chaurasiya, Jianming Lu, Anthony K. Park, Yanghee Woo, Susanne G. Warner, Yuman Fong and Nanhai G. Chen
    Citation: Journal of Translational Medicine 2018 16:110
  9. Saethre–Chotzen syndrome is an autosomal dominantly inherited disorder caused by mutations in the twist family basic helix-loop-helix transcription factor 1 (TWIST1) gene. Surgical procedures are frequently re...

    Authors: Shanshan Bai, Dong Li, Liang Xu, Huichuan Duan, Jie Yuan and Min Wei
    Citation: Journal of Translational Medicine 2018 16:103
  10. Radiation-induced lung toxicity (RILT) is a severe complication of radiotherapy in patients with thoracic tumors. Through proteomics, we have previously identified vitronectin (VTN) as a potential biomarker fo...

    Authors: Tian-Le Shen, Mi-Na Liu, Qin Zhang, Wen Feng, Wen Yu, Xiao-Long Fu and Xu-Wei Cai
    Citation: Journal of Translational Medicine 2018 16:100
  11. Members in Motin family, or Angiomotins (AMOTs), are adaptor proteins that localize in the membranous, cytoplasmic or nuclear fraction in a cell context-dependent manner. They control the bioprocesses such as ...

    Authors: Tingting Huang, Yuhang Zhou, Jinglin Zhang, Alfred S. L. Cheng, Jun Yu, Ka Fai To and Wei Kang
    Citation: Journal of Translational Medicine 2018 16:98
  12. Bone marrow stromal cells (BMSCs) have classically been cultured in media supplemented with fetal bovine serum (FBS). As an alternative to FBS, pooled solvent detergent apheresis platelets, HPGF-C18, was evalu...

    Authors: Jiaqiang Ren, Dawn Ward, Steven Chen, Katherine Tran, Ping Jin, Marianna Sabatino, Pamela G. Robey and David F. Stroncek
    Citation: Journal of Translational Medicine 2018 16:65
  13. Age-related macular degeneration (AMD) is a leading cause of blindness among the elderly characterized by retinal pigment epithelium (RPE) degeneration with accumulation of abnormal intracellular deposits (lip...

    Authors: George Inana, Christopher Murat, Weijun An, Xiang Yao, Ian R. Harris and Jing Cao
    Citation: Journal of Translational Medicine 2018 16:63
  14. Multiple sclerosis (MS) is a progressively debilitating neurological condition in which the immune system abnormally erodes the myelin sheath insulating the nerves. Mesenchymal stem cells (MSC) have been used ...

    Authors: Neil H. Riordan, Isabela Morales, Giselle Fernández, Nicole Allen, Neal E. Fearnot, Michael E. Leckrone, Dedra Jones Markovich, Darla Mansfield, Dorita Avila, Amit N. Patel, Santosh Kesari and Jorge Paz Rodriguez
    Citation: Journal of Translational Medicine 2018 16:57

    The Correction to this article has been published in Journal of Translational Medicine 2021 19:197

  15. Kidneys from deceased donors are being used to meet the growing need for grafts. However, delayed graft function (DGF) and acute rejection incidences are high, leading to adverse effects on graft outcomes. Opt...

    Authors: Qipeng Sun, Zhengyu Huang, Fei Han, Ming Zhao, Ronghua Cao, Daqiang Zhao, Liangqing Hong, Ning Na, Heng Li, Bin Miao, Jianmin Hu, Fanhang Meng, Yanwen Peng and Qiquan Sun
    Citation: Journal of Translational Medicine 2018 16:52
  16. Cell therapies are being investigated as potential disease modifying treatment options for osteoarthritis (OA). Progenza (PRG) comprises in vitro expanded mesenchymal stem cells derived from human donor adipos...

    Authors: D. Kuah, S. Sivell, T. Longworth, K. James, A. Guermazi, F. Cicuttini, Y. Wang, S. Craig, G. Comin, D. Robinson and J. Wilson
    Citation: Journal of Translational Medicine 2018 16:49
  17. The role of transforming growth factorβ (TGF-β)-induced tumor progression in advanced malignancy is well established, but the involvement of long non-coding RNAs (lncRNAs) in TGF-β signaling remains unclear. T...

    Authors: Teng Huang, Wei Huang, Hong Lu, Bi-yun Zhang, Jun Ma, Di Zhao, Yi-jun Wang, Da-hai Yu and Xia He
    Citation: Journal of Translational Medicine 2018 16:46
  18. Critical tissue defects frequently result from trauma, burns, chronic wounds and/or surgery. The ideal treatment for such tissue loss is autografting, but donor sites are often limited. Tissue engineering (TE)...

    Authors: Pan Wu, Haojiao Chen, Ronghua Jin, Tingting Weng, Jon Kee Ho, Chuangang You, Liping Zhang, Xingang Wang and Chunmao Han
    Citation: Journal of Translational Medicine 2018 16:29
  19. We have recently shown that memory B cells from murine CMV immune donor animals adoptively transferred into immunodeficient mice were highly effective in protecting from a viral infection indicating a therapeu...

    Authors: Hannes Tittlbach, Andrea Schneider, Julian Strobel, Robert Zimmermann, Stefanie Maas, Bernd Gebhardt, Georg Rauser, Michael Mach, Andreas Mackensen, Thomas H. Winkler and Julia Winkler
    Citation: Journal of Translational Medicine 2017 15:228
  20. Degenerative disc disease (DDD) is a common cause of lower back pain with radicular symptoms and has a significant socioeconomic impact given the associated disability. Limited effective conservative therapeut...

    Authors: Christopher Centeno, Jason Markle, Ehren Dodson, Ian Stemper, Christopher J. Williams, Matthew Hyzy, Thomas Ichim and Michael Freeman
    Citation: Journal of Translational Medicine 2017 15:197
  21. Neurotrophins (NT) belongs to a family of growth factors which promotes neurons survival and differentiation. Increasing evidence show that NT and their receptor are expressed in lung tissues suggesting a poss...

    Authors: Emanuela Cherubini, Salvatore Mariotta, Davide Scozzi, Rita Mancini, Giorgia Osman, Michela D’Ascanio, Pierdonato Bruno, Giuseppe Cardillo and Alberto Ricci
    Citation: Journal of Translational Medicine 2017 15:196
  22. This review based on translational research predicts that the transcription factor p53 is the key effector of all anti-acne therapies. All-trans retinoic acid (ATRA) and isotretinoin (13-cis retinoic acid) enhanc...

    Authors: Bodo C. Melnik
    Citation: Journal of Translational Medicine 2017 15:195
  23. Reduced levels of endothelial progenitor cells (EPCs) counts have been reported in diabetic mellitus (DM) patients and other diabetes-related disorder. EPCs are a circulating, bone marrow-derived cell populati...

    Authors: Rashmi K. Ambasta, Harleen Kohli and Pravir Kumar
    Citation: Journal of Translational Medicine 2017 15:185
  24. In humans, the heterochronic cascade composed of the RNA-binding protein LIN28 and its major target, the let-7 family of microRNAs (miRNAs), is highly regulated during human erythroid ontogeny. Additionally, down...

    Authors: Jaira F. de Vasconcellos, Colleen Byrnes, Y. Terry Lee, Joshua M. Allwardt, Megha Kaushal, Antoinette Rabel and Jeffery L. Miller
    Citation: Journal of Translational Medicine 2017 15:169
  25. Cardiovascular disease remains a major health care challenge. The knowledge about the underlying mechanisms of the respective vascular disease etiologies has greatly expanded over the last decades. This includ...

    Authors: Christoph S. Nabzdyk, Leena Pradhan-Nabzdyk and Frank W. LoGerfo
    Citation: Journal of Translational Medicine 2017 15:164
  26. In an attempt to increase the therapeutic potential for myocardial regeneration, there is a quest for new cell sources and types for cell therapy protocols. The pathophysiology of heart diseases may affect cel...

    Authors: Lucinara Dadda Dias, Karina Rabello Casali, Carine Ghem, Melissa Kristocheck da Silva, Grasiele Sausen, Patrícia Bonini Palma, Dimas Tadeu Covas, Renato A. K. Kalil, Beatriz D. Schaan, Nance Beyer Nardi and Melissa Medeiros Markoski
    Citation: Journal of Translational Medicine 2017 15:161
  27. Cell-based therapy is being explored as an alternative treatment option for critical limb ischemia (CLI), a disease associated with high amputation and mortality rates and poor quality of life. However, therap...

    Authors: Adas Darinskas, Mindaugas Paskevicius, Gintaras Apanavicius, Gintaris Vilkevicius, Liutauras Labanauskas, Thomas E. Ichim and Rytis Rimdeika
    Citation: Journal of Translational Medicine 2017 15:143

    The Retraction Note to this article has been published in Journal of Translational Medicine 2020 18:90

  28. Children with congenital heart defects (CHDs) are at high risk for myocardial failure after operative procedures with cardiopulmonary bypass (CPB). Recent studies suggest that microRNAs (miRNA) are involved in...

    Authors: Masood Abu-Halima, Martin Poryo, Nicole Ludwig, Janine Mark, Ina Marsollek, Christian Giebels, Johannes Petersen, Hans-Joachim Schäfers, Ulrich Grundmann, Thomas Pickardt, Andreas Keller, Eckart Meese and Hashim Abdul-Khaliq
    Citation: Journal of Translational Medicine 2017 15:117
  29. Numerous studies have investigated the role of the dietary factors in the prevention of cognitive decline but the short-term effects of foods choice on cognitive performances in the elderly are poorly explored...

    Authors: Elisa Mazza, Antonietta Fava, Yvelise Ferro, Marta Moraca, Stefania Rotundo, Carmela Colica, Francesco Provenzano, Rosa Terracciano, Marta Greco, Daniela Foti, Elio Gulletta, Diego Russo, Domenico Bosco, Arturo Pujia and Tiziana Montalcini
    Citation: Journal of Translational Medicine 2017 15:109
  30. Retinal degeneration (RD), such as age-related macular degeneration (AMD) and retinitis pigmentosa, is one of the leading causes of blindness. Presently, no satisfactory therapeutic options are available for t...

    Authors: Zhimin Tang, Yi Zhang, Yuyao Wang, Dandan Zhang, Bingqiao Shen, Min Luo and Ping Gu
    Citation: Journal of Translational Medicine 2017 15:99
  31. Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. However, these pivotal investigations have als...

    Authors: David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava and Roland W. Herzog
    Citation: Journal of Translational Medicine 2017 15:94
  32. The use of fetal bovine serum (FBS) as a media supplement for the ex vivo expansion of bone-marrow derived mesenchymal stromal cells (BM-MSC) has been discouraged by regulatory agencies, due to the risk of tra...

    Authors: Martina Bernardi, Francesco Agostini, Katia Chieregato, Eliana Amati, Cristina Durante, Mario Rassu, Marco Ruggeri, Sabrina Sella, Elisabetta Lombardi, Mario Mazzucato and Giuseppe Astori
    Citation: Journal of Translational Medicine 2017 15:90
  33. Hepatocellular carcinoma (HCC) is one of the most common cancers in China and frequently occurs with chronic hepatitis B virus infection. To investigate whether cell-based cancer immunotherapy induces tumor sp...

    Authors: Yanyan Han, Yeting Wu, Chou Yang, Jing Huang, Yabing Guo, Li Liu, Ping Chen, Dongyun Wu, Junyun Liu, Jin Li, Xiangjun Zhou and Jinlin Hou
    Citation: Journal of Translational Medicine 2017 15:64
  34. Clinical trials of Chimeric Antigen Receptor (CAR) T cells manufactured from autologous peripheral blood mononuclear cell (PBMC) concentrates for the treatment of hematologic malignancies have been promising, ...

    Authors: David F. Stroncek, Daniel W. Lee, Jiaqiang Ren, Marianna Sabatino, Steven Highfill, Hanh Khuu, Nirali N. Shah, Rosandra N. Kaplan, Terry J. Fry and Crystal L. Mackall
    Citation: Journal of Translational Medicine 2017 15:59
  35. Critical limb ischemia (CLI) causes severe ischemic rest pain, ulcer, and gangrene in the lower limbs. In spite of angioplasty and surgery, CLI patients without suitable artery inflow or enough vascular bed in...

    Authors: Makoto Samura, Tohru Hosoyama, Yuriko Takeuchi, Koji Ueno, Noriyasu Morikage and Kimikazu Hamano
    Citation: Journal of Translational Medicine 2017 15:49
  36. Bone marrow mesenchymal stem cells (BMMSCs) and bone marrow mononuclear cells (BMMNCs) are both used to treat spastic cerebral palsy. However, the differences in therapeutic effect remain unknown.

    Authors: Xuebin Liu, Xiaojun Fu, Guanghui Dai, Xiaodong Wang, Zan Zhang, Hongbin Cheng, Pei Zheng and Yihua An
    Citation: Journal of Translational Medicine 2017 15:48
  37. Sarcopenia, osteoporosis and osteoarthritis are the most frequent musculoskeletal disorders affecting older people. The main aim of this study was to test the hypothesis that the balance between BMPs and myost...

    Authors: Manuel Scimeca, Eleonora Piccirilli, Francesca Mastrangeli, Cecilia Rao, Maurizio Feola, Augusto Orlandi, Elena Gasbarra, Elena Bonanno and Umberto Tarantino
    Citation: Journal of Translational Medicine 2017 15:34
  38. Mesenchymal stem cell (MSC) has been widely explored in the past decade as a cell-based treatment for various diseases. However, poor survival of adaptively transferred MSCs limits their clinical therapeutic p...

    Authors: Shuo Wang, Chao Zhang, Sidikejiang Niyazi, Long Zheng, Jiawei Li, Weitao Zhang, Ming Xu, Ruiming Rong, Cheng Yang and Tongyu Zhu
    Citation: Journal of Translational Medicine 2017 15:33
  39. We performed a randomized, double-blind, cross-over study to assess the neuroregenerative potential of intravenous granulocyte colony-stimulating factor (G-CSF) followed by infusion of mobilized peripheral blo...

    Authors: Wee-Jin Rah, Young-Ho Lee, Jin-Hwa Moon, Hyun-Ju Jun, Hye-Ryeong Kang, Hani Koh, Hye Jung Eom, Ji Young Lee, Young Jun Lee, Ji Young Kim, Yun-Young Choi, Kyeongil Park, Mi Jung Kim and Seung-Hyun Kim
    Citation: Journal of Translational Medicine 2017 15:16
  40. Stromal vascular fraction (SVF) can easily be obtained from a mini-lipoaspirate procedure of fat tissue and platelet rich plasma (PRP) can be obtained from peripheral blood. The SVF contains a mixture of cells...

    Authors: Kristin Comella, Robert Silbert and Michelle Parlo
    Citation: Journal of Translational Medicine 2017 15:12

    The Erratum to this article has been published in Journal of Translational Medicine 2017 15:108

  41. The repair of urinary bladder tissue is a necessity for tissue loss due to cancer, trauma, or congenital abnormalities. Use of intestinal tissue is still the gold standard in the urological clinic, which leads...

    Authors: Dorothea Leonhäuser, Katja Stollenwerk, Volker Seifarth, Isabella M. Zraik, Michael Vogt, Pramod K. Srinivasan, Rene H. Tolba and Joachim O. Grosse
    Citation: Journal of Translational Medicine 2017 15:3
  42. Induced pluripotent stem cells (iPSC) possess an enormous potential as both, scientific and therapeutic tools. Their application in the regenerative medicine provides new treatment opportunities for numerous d...

    Authors: Maciej P. Walczak, Anna M. Drozd, Ewelina Stoczynska-Fidelus, Piotr Rieske and Dawid P. Grzela
    Citation: Journal of Translational Medicine 2016 14:341
  43. Recent studies suggest that immunotherapy using T regulatory cells (Tregs) prolongs remission in type 1 diabetes (T1DM). Here, we report factors that possibly affect the efficacy of this treatment.

    Authors: Natalia Marek-Trzonkowska, Małgorzata Myśliwiec, Dorota Iwaszkiewicz-Grześ, Mateusz Gliwiński, Ilona Derkowska, Magdalena Żalińska, Maciej Zieliński, Marcelina Grabowska, Hanna Zielińska, Karolina Piekarska, Anna Jaźwińska-Curyłło, Radosław Owczuk, Agnieszka Szadkowska, Krystyna Wyka, Piotr Witkowski, Wojciech Młynarski…
    Citation: Journal of Translational Medicine 2016 14:332
  44. Myocardial recovery with left ventricular assist device (LVAD) therapy is highly variable and difficult to predict. Next generation ribonucleic acid (RNA) sequencing is an innovative, rapid, and quantitative a...

    Authors: Kajari Dhar, Alexandra M. Moulton, Eric Rome, Fang Qiu, Jeff Kittrell, Eugenia Raichlin, Ronald Zolty, John Y. Um, Michael J. Moulton, Hesham Basma, Daniel R. Anderson, James D. Eudy and Brian D. Lowes
    Citation: Journal of Translational Medicine 2016 14:327
  45. Adipose derived stromal cells (ASCs) are a rich and convenient source of cells for clinical regenerative therapeutic approaches. However, applications of ASCs often require cell expansion to reach the needed d...

    Authors: Mandana Haack-Sørensen, Bjarke Follin, Morten Juhl, Sonja K. Brorsen, Rebekka H. Søndergaard, Jens Kastrup and Annette Ekblond
    Citation: Journal of Translational Medicine 2016 14:319
  46. Cell-based therapies such as autologous chondrocyte implantation are promising therapeutic approaches to treat cartilage defects to prevent further cartilage degeneration. To assure consistent quality of cell-...

    Authors: Christoph Bartz, Miriam Meixner, Petra Giesemann, Giulietta Roël, Grit-Carsta Bulwin and Jeske J. Smink
    Citation: Journal of Translational Medicine 2016 14:317
  47. The endometrial regenerative cell (ERC) is a novel type of adult mesenchymal stem cell isolated from menstrual blood. Previous studies demonstrated that ERCs possess unique immunoregulatory properties in vitro...

    Authors: Shanzheng Lu, Ganggang Shi, Xiaoxi Xu, Grace Wang, Xu Lan, Peng Sun, Xiang Li, Baoren Zhang, Xiangying Gu, Thomas E. Ichim and Hao Wang
    Citation: Journal of Translational Medicine 2016 14:300
  48. Gene therapy protocols require robust and long-term gene expression. For two decades, retrovirus family vectors have offered several attractive properties as stable gene-delivery vehicles. These vectors repres...

    Authors: José Eduardo Vargas, Leonardo Chicaybam, Renato Tetelbom Stein, Amilcar Tanuri, Andrés Delgado-Cañedo and Martin H. Bonamino
    Citation: Journal of Translational Medicine 2016 14:288

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