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.

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  1. Research

    A novel cytoprotective peptide protects mesenchymal stem cells against mitochondrial dysfunction and apoptosis induced by starvation via Nrf2/Sirt3/FoxO3a pathway

    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...

    Shuo Wang, Chao Zhang, Sidikejiang Niyazi, Long Zheng, Jiawei Li, Weitao Zhang, Ming Xu, Ruiming Rong, Cheng Yang and Tongyu Zhu

    Journal of Translational Medicine 2017 15:33

    Published on: 15 February 2017

  2. Research

    Bone Morphogenetic Proteins and myostatin pathways: key mediator of human sarcopenia

    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...

    Manuel Scimeca, Eleonora Piccirilli, Francesca Mastrangeli, Cecilia Rao, Maurizio Feola, Augusto Orlandi, Elena Gasbarra, Elena Bonanno and Umberto Tarantino

    Journal of Translational Medicine 2017 15:34

    Published on: 15 February 2017

  3. Research

    Neuroregenerative potential of intravenous G-CSF and autologous peripheral blood stem cells in children with cerebral palsy: a randomized, double-blind, cross-over study

    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...

    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

    Journal of Translational Medicine 2017 15:16

    Published on: 21 January 2017

  4. Research

    Effects of the intradiscal implantation of stromal vascular fraction plus platelet rich plasma in patients with degenerative disc disease

    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...

    Kristin Comella, Robert Silbert and Michelle Parlo

    Journal of Translational Medicine 2017 15:12

    Published on: 13 January 2017

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

  5. Research

    Two differentially structured collagen scaffolds for potential urinary bladder augmentation: proof of concept study in a Göttingen minipig model

    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...

    Dorothea Leonhäuser, Katja Stollenwerk, Volker Seifarth, Isabella M. Zraik, Michael Vogt, Pramod K. Srinivasan, Rene H. Tolba and Joachim O. Grosse

    Journal of Translational Medicine 2017 15:3

    Published on: 4 January 2017

  6. Research

    Directed differentiation of human iPSC into insulin producing cells is improved by induced expression of PDX1 and NKX6.1 factors in IPC progenitors

    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...

    Maciej P. Walczak, Anna M. Drozd, Ewelina Stoczynska-Fidelus, Piotr Rieske and Dawid P. Grzela

    Journal of Translational Medicine 2016 14:341

    Published on: 20 December 2016

  7. Research

    Factors affecting long-term efficacy of T regulatory cell-based therapy in type 1 diabetes

    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.

    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…

    Journal of Translational Medicine 2016 14:332

    Published on: 1 December 2016

  8. Research

    Targeted myocardial gene expression in failing hearts by RNA sequencing

    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...

    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

    Journal of Translational Medicine 2016 14:327

    Published on: 25 November 2016

  9. Research

    Culture expansion of adipose derived stromal cells. A closed automated Quantum Cell Expansion System compared with manual flask-based culture

    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...

    Mandana Haack-Sørensen, Bjarke Follin, Morten Juhl, Sonja K. Brorsen, Rebekka H. Søndergaard, Jens Kastrup and Annette Ekblond

    Journal of Translational Medicine 2016 14:319

    Published on: 16 November 2016

  10. Research

    An ex vivo human cartilage repair model to evaluate the potency of a cartilage cell transplant

    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-...

    Christoph Bartz, Miriam Meixner, Petra Giesemann, Giulietta Roël, Grit-Carsta Bulwin and Jeske J. Smink

    Journal of Translational Medicine 2016 14:317

    Published on: 15 November 2016

  11. Research

    Human endometrial regenerative cells alleviate carbon tetrachloride-induced acute liver injury in mice

    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...

    Shanzheng Lu, Ganggang Shi, Xiaoxi Xu, Grace Wang, Xu Lan, Peng Sun, Xiang Li, Baoren Zhang, Xiangying Gu, Thomas E. Ichim and Hao Wang

    Journal of Translational Medicine 2016 14:300

    Published on: 22 October 2016

  12. Review

    Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives

    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...

    José Eduardo Vargas, Leonardo Chicaybam, Renato Tetelbom Stein, Amilcar Tanuri, Andrés Delgado-Cañedo and Martin H. Bonamino

    Journal of Translational Medicine 2016 14:288

    Published on: 12 October 2016

  13. Research

    Intra-discal injection of autologous, hypoxic cultured bone marrow-derived mesenchymal stem cells in five patients with chronic lower back pain: a long-term safety and feasibility study

    Chronic low back pain due to disc degeneration represents a major social and economic burden worldwide. The current standard of care is limited to symptomatic relief and no current approved therapy promotes di...

    Christian Elabd, Christopher J. Centeno, John R. Schultz, Gregory Lutz, Thomas Ichim and Francisco J. Silva

    Journal of Translational Medicine 2016 14:253

    Published on: 1 September 2016

  14. Research

    Matrix- and plasma-derived peptides promote tissue-specific injury responses and wound healing in diabetic swine

    Non-healing wounds are a major global health concern and account for the majority of non-traumatic limb amputations worldwide. However, compared to standard care practices, few advanced therapeutics effectivel...

    Anthony R. Sheets, Conner J. Massey, Stephen M. Cronk, Mark D. Iafrati and Ira M. Herman

    Journal of Translational Medicine 2016 14:197

    Published on: 2 July 2016

  15. Research

    Effects of the intramyocardial implantation of stromal vascular fraction in patients with chronic ischemic cardiomyopathy

    Stromal vascular fraction (SVF) can easily be obtained from a mini-lipoaspirate procedure of fat tissue. The SVF contains a mixture of cells including ADSCs and growth factors and has been depleted of the adip...

    K. Comella, J. Parcero, H. Bansal, J. Perez, J. Lopez, A. Agrawal and T. Ichim

    Journal of Translational Medicine 2016 14:158

    Published on: 2 June 2016

  16. Research

    Clinical-scale expansion of mesenchymal stromal cells: a large banking experience

    Mesenchymal stromal cells (MSC) are largely investigated in clinical trials aiming to control inappropriate immune reactions (GVHD, Crohn’s disease, solid organ transplantation). As the percentage of MSC precu...

    Chantal Lechanteur, Alexandra Briquet, Olivier Giet, Olivier Delloye, Etienne Baudoux and Yves Beguin

    Journal of Translational Medicine 2016 14:145

    Published on: 20 May 2016

  17. Research

    Finding a new therapeutic approach for no-option Parkinsonisms: mesenchymal stromal cells for progressive supranuclear palsy

    The trophic, anti-apoptotic and regenerative effects of bone marrow mesenchymal stromal cells (MSC) may reduce neuronal cell loss in neurodegenerative disorders.

    Margherita Canesi, Rosaria Giordano, Lorenza Lazzari, Maurizio Isalberti, Ioannis Ugo Isaias, Riccardo Benti, Paolo Rampini, Giorgio Marotta, Aurora Colombo, Emanuele Cereda, Mariangela Dipaola, Tiziana Montemurro, Mariele Viganò, Silvia Budelli, Elisa Montelatici, Cristiana Lavazza…

    Journal of Translational Medicine 2016 14:127

    Published on: 10 May 2016

  18. Research

    Suction assisted liposuction does not impair the regenerative potential of adipose derived stem cells

    Adipose-derived stem cells (ASCs) have been identified as a population of multipotent cells with promising applications in tissue engineering and regenerative medicine. ASCs are abundant in fat tissue, which c...

    Dominik Duscher, Anna Luan, Robert C. Rennert, David Atashroo, Zeshaan N. Maan, Elizabeth A. Brett, Alexander J. Whittam, Natalie Ho, Michelle Lin, Michael S. Hu, Graham G. Walmsley, Raphael Wenny, Manfred Schmidt, Arndt F. Schilling, Hans-Günther Machens, Georg M. Huemer…

    Journal of Translational Medicine 2016 14:126

    Published on: 6 May 2016

  19. Review

    Safety of targeting tumor endothelial cell antigens

    The mechanisms underlying discrimination between “self” and “non-self”, a central immunological principle, require careful consideration in immune oncology therapeutics where eliciting anti-cancer immunity mus...

    Samuel C. Wagner, Neil H. Riordan, Thomas E. Ichim, Julia Szymanski, Hong Ma, Jesus A. Perez, Javier Lopez, Juan J. Plata-Munoz, Francisco Silva, Amit N. Patel and Santosh Kesari

    Journal of Translational Medicine 2016 14:90

    Published on: 12 April 2016

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