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

    Stromal vascular fraction cells for the treatment of critical limb ischemia: a pilot study

    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...

    Adas Darinskas, Mindaugas Paskevicius, Gintaras Apanavicius, Gintaris Vilkevicius, Liutauras Labanauskas, Thomas E. Ichim and Rytis Rimdeika

    Journal of Translational Medicine 2017 15:143

    Published on: 19 June 2017

  2. Research

    Intra-articular injection in the knee of adipose derived stromal cells (stromal vascular fraction) and platelet rich plasma for osteoarthritis

    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. We evaluated the safety and prelimi...

    Himanshu Bansal, Kristin Comella, Jerry Leon, Poonam Verma, Diwaker Agrawal, Prasad Koka and Thomas Ichim

    Journal of Translational Medicine 2017 15:141

    Published on: 19 June 2017

  3. Research

    Differential expression of microRNAs following cardiopulmonary bypass in children with congenital heart diseases

    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...

    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

    Journal of Translational Medicine 2017 15:117

    Published on: 30 May 2017

  4. Research

    Impact of legumes and plant proteins consumption on cognitive performances in the elderly

    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...

    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

    Journal of Translational Medicine 2017 15:109

    Published on: 22 May 2017

  5. Review

    Progress of stem/progenitor cell-based therapy for retinal degeneration

    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...

    Zhimin Tang, Yi Zhang, Yuyao Wang, Dandan Zhang, Bingqiao Shen, Min Luo and Ping Gu

    Journal of Translational Medicine 2017 15:99

    Published on: 10 May 2017

  6. Research

    The production method affects the efficacy of platelet derivatives to expand mesenchymal stromal cells in vitro

    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...

    Martina Bernardi, Francesco Agostini, Katia Chieregato, Eliana Amati, Cristina Durante, Mario Rassu, Marco Ruggeri, Sabrina Sella, Elisabetta Lombardi, Mario Mazzucato and Giuseppe Astori

    Journal of Translational Medicine 2017 15:90

    Published on: 1 May 2017

  7. Research

    Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

    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...

    David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava and Roland W. Herzog

    Journal of Translational Medicine 2017 15:94

    Published on: 1 May 2017

  8. Research

    Dynamic and specific immune responses against multiple tumor antigens were elicited in patients with hepatocellular carcinoma after cell-based immunotherapy

    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...

    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

    Journal of Translational Medicine 2017 15:64

    Published on: 22 March 2017

  9. Research

    Elutriated lymphocytes for manufacturing chimeric antigen receptor T cells

    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, ...

    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

    Journal of Translational Medicine 2017 15:59

    Published on: 16 March 2017

  10. Review

    Therapeutic strategies for cell-based neovascularization in critical limb ischemia

    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...

    Makoto Samura, Tohru Hosoyama, Yuriko Takeuchi, Koji Ueno, Noriyasu Morikage and Kimikazu Hamano

    Journal of Translational Medicine 2017 15:49

    Published on: 24 February 2017

  11. Research

    Comparative analysis of curative effect of bone marrow mesenchymal stem cell and bone marrow mononuclear cell transplantation for spastic cerebral palsy

    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.

    Xuebin Liu, Xiaojun Fu, Guanghui Dai, Xiaodong Wang, Zan Zhang, Hongbin Cheng, Pei Zheng and Yihua An

    Journal of Translational Medicine 2017 15:48

    Published on: 24 February 2017

  12. 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

  13. 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

  14. 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

  15. 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

  16. 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

  17. 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

  18. 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

  19. 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

  20. 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

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