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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. The colorectum includes ascending colon, transverse colon, descending colon, sigmoid colon, and rectum. Different sites of colorectal cancer (CRC) are different in many aspects, including clinical symptoms, bi...

    Authors: Yang Xi, Pan Yuefen, Wu Wei, Qi Quan, Zhuang Jing, Xu Jiamin and Han Shuwen
    Citation: Journal of Translational Medicine 2019 17:353
    Content type: Research Published on:

  2. Liver cancer is the second leading causes of cancer-related death globally. Pyrroline-5-carboxylate reductase 1 (PYCR1) plays a critical role in metabolic profiles of tumors. Therefore, it is necessary to expl...

    Authors: Juhua Zhuang, Yanan Song, Ying Ye, Saifei He, Xing Ma, Miao Zhang, Jing Ni, Jiening Wang and Wei Xia
    Citation: Journal of Translational Medicine 2019 17:343
    Content type: Research Published on:

  3. The P38 mitogen-activated protein kinase (MAPK) pathway plays an essential role in CVB3-induced diseases. We previously demonstrated microRNA-21 has potential inhibitory effect on the MAP2K3 which locates upst...

    Authors: Feng He, Zonghui Xiao, Hailan Yao, Sen Li, Miao Feng, Wei Wang, Zhewei Liu, Zhuo Liu and Jianxin Wu
    Citation: Journal of Translational Medicine 2019 17:335
    Content type: Research Published on:

  4. Mesenchymal stem cells (MSCs) are attractive cell-therapy candidates. Despite their popularity and promise, there is no uniform method of preparation of MSCs. Typically, cells are cryopreserved in liquid nitro...

    Authors: Ben Antebi, Amber M. Asher, Luis A. Rodriguez II, Robbie K. Moore, Arezoo Mohammadipoor and Leopoldo C. Cancio
    Citation: Journal of Translational Medicine 2019 17:297
    Content type: Research Published on:

  5. Glutathione S-transferase A3 (GSTA3) is known as an antioxidative protease, however, the crucial role of GSTA3 in liver fibrosis remains unclear. As a recently we developed water-soluble pyridone agent with antif...

    Authors: Haihua Chen, Qixin Gan, Congying Yang, Xiongqun Peng, Jiao Qin, Sisi Qiu, Yanzhi Jiang, Sha Tu, Ying He, Shenglan Li, Huixiang Yang, Lijian Tao and Yu Peng
    Citation: Journal of Translational Medicine 2019 17:280
    Content type: Research Published on:

    The Correction to this article has been published in Journal of Translational Medicine 2020 18:182

  6. Deep venous thrombosis (DVT) of lower extremities is a common thrombotic disease, occurring either in isolation or as a complication of other diseases or procedures. MiR-21 is one of important microRNAs which ...

    Authors: Xiaolong Du, Lei Hong, Lili Sun, Hongfei Sang, Aiming Qian, Wendong Li, Hao Zhuang, Huoqi Liang, Dandan Song, Chenglong Li, Wenbin Wang and Xiaoqiang Li
    Citation: Journal of Translational Medicine 2019 17:270
    Content type: Research Published on:

  7. Mesenchymal stromal cells (MSC) demonstrate innate and regulatory immunologic functions and have been widely explored for cell therapy applications. Mechanisms by which MSCs achieve therapeutic effects are the...

    Authors: Alexandra Burr and Biju Parekkadan
    Citation: Journal of Translational Medicine 2019 17:263
    Content type: Research Published on:

  8. Cell therapy has been proposed for patients with critical limb ischemia (CLI). Autologous bone marrow derived cells (BMCs) have been mostly used, mesenchymal stem cells (MSCs) being an alternative. The aim of ...

    Authors: Rida Al-Rifai, Philippe Nguyen, Nicole Bouland, Christine Terryn, Lukshe Kanagaratnam, Gaël Poitevin, Caroline François, Catherine Boisson-Vidal, Marie-Antoinette Sevestre and Claire Tournois
    Citation: Journal of Translational Medicine 2019 17:261
    Content type: Research Published on:

  9. The rapid evolution of cell-based immunotherapies such as chimeric antigen receptor T-cells for treatment of hematological cancers has precipitated the need for a platform to expand these cells ex vivo in a sa...

    Authors: Claire Coeshott, Boah Vang, Mark Jones and Brian Nankervis
    Citation: Journal of Translational Medicine 2019 17:258
    Content type: Research Published on:

  10. Diabetic cardiomyopathy (DCM) is a common complication of diabetes and is characterized by chronic myocardial inflammation. Mesenchymal stem cell (MSC) infusions have recently been suggested to alleviate myoca...

    Authors: Liyuan Jin, Zihui Deng, Jinying Zhang, Chen Yang, Jiejie Liu, Weidong Han, Ping Ye, Yiling Si and Guanghui Chen
    Citation: Journal of Translational Medicine 2019 17:251
    Content type: Research Published on:

  11. Here, we isolated, expanded and functionally characterized regulatory T cells (Tregs) from patients with end stage kidney and liver disease, waiting for kidney/liver transplantation (KT/LT), with the aim to es...

    Authors: Francesca Ulbar, Tiziana Montemurro, Tatiana Jofra, Miriam Capri, Giorgia Comai, Valentina Bertuzzo, Cristiana Lavazza, Alessandra Mandelli, Mariele Viganò, Silvia Budelli, Maria Giulia Bacalini, Chiara Pirazzini, Paolo Garagnani, Valeria Giudice, Daria Sollazzo, Antonio Curti…
    Citation: Journal of Translational Medicine 2019 17:250
    Content type: Research Published on:

  12. The relationship between the tissue injury healing response and development of heterotopic ossification (HO) is poorly understood. Here we compare a rat blast model and human traumatized muscle from a blast in...

    Authors: Jaira F. de Vasconcellos, Sonia Zicari, Stephen D. Fernicola, Daniel W. Griffin, Youngmi Ji, Emily H. Shin, Patrick Jones, Gregory T. Christopherson, Husain Bharmal, Carl Cirino, Thao Nguyen, Astor Robertson, Vincent D. Pellegrini Jr. and Leon J. Nesti
    Citation: Journal of Translational Medicine 2019 17:248
    Content type: Research Published on:

  13. The molecular pathways that drive bone marrow myeloid progenitors (BMMP) development are very well understood and include a tight controlled multi-stage gene hierarch. Monocytes are versatile cells that displa...

    Authors: Mariana Cabanel, Thayse Pinheiro da Costa, Marcia Cury EL-Cheikh and Katia Carneiro
    Citation: Journal of Translational Medicine 2019 17:247
    Content type: Research Published on:

  14. Cell-based therapies have the potential to become treatment options for many diseases, but efficient scale-out of these therapies has proven to be a major hurdle. Bioreactors can be used to overcome this hurdl...

    Authors: Ruud Das, Rens Roosloot, Melissa van Pel, Koen Schepers, Marijn Driessen, Willem E. Fibbe, Joost Dick de Bruijn and Helene Roelofs
    Citation: Journal of Translational Medicine 2019 17:241
    Content type: Research Published on:

  15. The use of “off-the-shelf” cellular therapy products derived from healthy donors addresses many of the challenges associated with customized cell products. However, the potential of allogeneic cell products to...

    Authors: David H. Quach, Luis Becerra-Dominguez, Rayne H. Rouce and Cliona M. Rooney
    Citation: Journal of Translational Medicine 2019 17:240
    Content type: Research Published on:

  16. The clinical significance of stem cell therapy in the treatment of dilated cardiomyopathy remains unclear. This systemic appraisal and meta-analysis aimed to assess the efficacy and safety of stem cell therapy...

    Authors: Shu-Ling Rong, Ze-Kun Wang, Xue-Dong Zhou, Xiao-Lin Wang, Zhi-Ming Yang and Bao Li
    Citation: Journal of Translational Medicine 2019 17:221
    Content type: Research Published on:

  17. Extracellular vesicles from bone marrow-derived multipotent mesenchymal stromal cells (BMSC-EVs) can play important roles in the repair of injured tissues. However, no reports have investigated the role and un...

    Authors: Zhengzhou Shi, Qi Wang and Dapeng Jiang
    Citation: Journal of Translational Medicine 2019 17:211
    Content type: Research Published on:

  18. “Nanomedicine” is the application of purposely designed nano-scale materials for improved therapeutic and diagnostic outcomes, which cannot be otherwise achieved using conventional delivery approaches. While “...

    Authors: Jie Feng, Chester E. Markwalter, Chang Tian, Madeleine Armstrong and Robert K. Prud’homme
    Citation: Journal of Translational Medicine 2019 17:200
    Content type: Research Published on:

  19. Telocytes play key roles in maintenance of organ/tissue function and prevention of organ injury. However, there are great challenges to investigate telocytes functions using primary telocytes, due to the diffi...

    Authors: Dongli Song, Menglin Xu, Ruixue Qi, Ruihua Ma, Yile Zhou, Duojiao Wu, Hao Fang and Xiangdong Wang
    Citation: Journal of Translational Medicine 2019 17:158
    Content type: Research Published on:

  20. Fulminant liver failure (FHF) is a serious clinical problem and liver transplantation is the major intervention. But the overall survival rate of FHF is low owing to the donated organ shortage. Apolipoprotein ...

    Authors: Ya-Chao Tao, Meng-Lan Wang, Dong-Bo Wu, Chen Luo, Hong Tang and En-Qiang Chen
    Citation: Journal of Translational Medicine 2019 17:151
    Content type: Research Published on:

  21. Interferon gamma (IFNγ) plays an important role in the development of chronic lung diseases via the production of inflammatory mediators, although the exact mechanism remains unclear. The present study aimed a...

    Authors: Yichun Zhu, Dongli Song, Yuanlin Song and Xiangdong Wang
    Citation: Journal of Translational Medicine 2019 17:147
    Content type: Research Published on:

  22. The role of γδ T cells, innate-like lymphocytes with unrestrained MHC, in various malignancies has recently been extensively studied. However, there is limited research regarding γδ T cells in ovarian cancer (...

    Authors: Xian Chen, Wenwen Shang, Rui Xu, Ming Wu, Xiaojie Zhang, Peijun Huang, Fang Wang and Shiyang Pan
    Citation: Journal of Translational Medicine 2019 17:144
    Content type: Research Published on:

  23. Mitochondria take part in a network of cellular processes that regulate cell homeostasis. Defects in mitochondrial function are key pathophysiological changes during acute kidney injury (AKI). Mesenchymal stem...

    Authors: Lingfei Zhao, Chenxia Hu, Ping Zhang, Hua Jiang and Jianghua Chen
    Citation: Journal of Translational Medicine 2019 17:142
    Content type: Review Published on:

  25. Cell based therapies, such as bone marrow derived mesenchymal stem cells (BM-MSCs; also known as mesenchymal stromal cells), are currently under investigation for a number of disease applications. The current ...

    Authors: Alpa Trivedi, Byron Miyazawa, Stuart Gibb, Kristen Valanoski, Lindsay Vivona, Maximillian Lin, Daniel Potter, Mars Stone, Philip J. Norris, James Murphy, Sawyer Smith, Martin Schreiber and Shibani Pati
    Citation: Journal of Translational Medicine 2019 17:128
    Content type: Research Published on:

  26. Cancer is a serious health issue in the world due to a large body of cancer-related human deaths, and there is no current treatment available to efficiently treat the disease as the tumor is often diagnosed at...

    Authors: Yanyan Han
    Citation: Journal of Translational Medicine 2019 17:116
    Content type: Review Published on:

  27. The number of Mesenchymal Stem/Stromal Cells (MSCs) in the human bone marrow (BM) is small compared to other cell types. BM aspirate concentration (BMAC) may be used to increase numbers of MSCs, but the compos...

    Authors: Richard Schäfer, Malcolm R. DeBaun, Erika Fleck, Christopher J. Centeno, Daniela Kraft, Johannes Leibacher, Karen Bieback, Erhard Seifried and Jason L. Dragoo
    Citation: Journal of Translational Medicine 2019 17:115
    Content type: Research Published on:

  28. Monoallelic expression (MAE) is a frequent genomic phenomenon in normal tissues, however its role in cancer is yet to be fully understood. MAE is defined as the expression of a gene that is restricted to one a...

    Authors: Lee Silcock, Hakeem Almabrazi, Younes Mokrab, Puthen Jithesh, Muna Al-Hashmi, Nicola James, Rebecca Mathew, Valentina Mattei, Davide Bedognetti, Francesca Lessi, Ramzi Temanni, Barbara Seliger, Rashid Al-Ali, Francesco M. Marincola, Ena Wang and Sara Tomei
    Citation: Journal of Translational Medicine 2019 17:112
    Content type: Research Published on:

  29. A stroke caused by angiostenosis always has a poor prognosis. Bone marrow stromal cells (BMSC) are widely applied in vascular regeneration. Recently, thrombospondin-4 (TSP4) was reported to promote the regener...

    Authors: Qian Zhang, Meiling Zhou, Xiangfeng Wu, Zhu Li, Bing Liu, Wenbin Gao, Jin Yue and Tao Liu
    Citation: Journal of Translational Medicine 2019 17:111
    Content type: Research Published on:

  30. Bone marrow-derived stem cells (BMSCs) and chondrocytes have been reported to present “dedifferentiation” and “phenotypic loss” during the chondrogenic differentiation process in cartilage tissue engineering, ...

    Authors: Ke Xue, Xiaodie Zhang, Zixu Gao, Wanyao Xia, Lin Qi and Kai Liu
    Citation: Journal of Translational Medicine 2019 17:104
    Content type: Research Published on:

  31. Monocytes are myeloid cells that reside in the blood and bone marrow and respond to inflammation. At the site of inflammation, monocytes express cytokines and chemokines. Monocytes have been shown to be cytoto...

    Authors: Daniel S. Green, Ana T. Nunes, Kevin W. Tosh, Virginia David-Ocampo, Vicki S. Fellowes, Jiaqiang Ren, Jianjian Jin, Sue-Ellen Frodigh, Chauha Pham, Jolynn Procter, Celina Tran, Irene Ekwede, Hanh Khuu, David F. Stroncek, Steven L. Highfill, Kathryn C. Zoon…
    Citation: Journal of Translational Medicine 2019 17:82
    Content type: Methodology Published on:

  32. Mesenchymal stromal cells (MSCs) were originally characterized by the ability to differentiate into different mesenchymal lineages in vitro, and their immunomodulatory and trophic functions have recently arous...

    Authors: Ya-nan Bie, Peng Gu, Yu-ting Chen, Xiao-xu Zhou, Yu-guang Tian, Qin Yang, Hai-yan Li, Xia Lin, Yan-hong Guan, Tao-yan Lin, Xun Lu, Hong-fen Shen, Ting-xiao Fang, Yu-min Liu, Dong Xiao and Wei-Wang Gu
    Citation: Journal of Translational Medicine 2019 17:72
    Content type: Research Published on:

  33. Human amniotic fluid (AF) contains numerous nutrients, trophic factors and defense proteins that provide a nurturing and protective environment for fetal development. Based on reports that AF has antibacterial...

    Authors: Yong Mao, Jan Pierce, Anya Singh-Varma, Michael Boyer, Joachim Kohn and Jo-Anna Reems
    Citation: Journal of Translational Medicine 2019 17:68
    Content type: Research Published on:

  34. As the optimal source of seed cells in periodontal tissue engineering, periodontal ligament stem cells (PDLSCs) have always been researched to improve cell expansion due to their limited resource and spontaneo...

    Authors: Chunshu Zhang, Hongmei Guo, Chengzhe Yang, Qian Chen, Jiahui Huang, Lianlian Liu, Yu Zhang, Shanshan Jin, Aimei Song and Pishan Yang
    Citation: Journal of Translational Medicine 2019 17:66
    Content type: Research Published on:

  35. Bone marrow stromal cells (BMSCs) are a heterogeneous population that participates in wound healing, immune modulation and tissue regeneration. Next generation sequencing was used to analyze transcripts from s...

    Authors: Shutong Liu, David F. Stroncek, Yingdong Zhao, Victoria Chen, Rongye Shi, Jinguo Chen, Jiaqiang Ren, Hui Liu, Hee Joon Bae, Steven L. Highfill and Ping Jin
    Citation: Journal of Translational Medicine 2019 17:23
    Content type: Research Published on:

  36. This study examined the quality of bone marrow aspirates extracted using a novel, FDA cleared method to optimally target cells from the inner cortical iliac bone surface without the need for centrifugation. Th...

    Authors: Michael Scarpone, Daniel Kuebler, Andrew Chambers, Carlo Maria De Filippo, Mariangela Amatuzio, Thomas E. Ichim, Amit N. Patel and Eugenio Caradonna
    Citation: Journal of Translational Medicine 2019 17:10
    Content type: Research Published on:

  37. Cell-based therapies have shown promise for the treatment of knee osteoarthritis (OA). The current study compared exercise therapy to autologous bone marrow concentrate (BMC) and platelet products for knee OA ...

    Authors: Christopher Centeno, Mitchell Sheinkop, Ehren Dodson, Ian Stemper, Christopher Williams, Matthew Hyzy, Thomas Ichim and Michael Freeman
    Citation: Journal of Translational Medicine 2018 16:355
    Content type: Research Published on:

  38. Adipose-derived mesenchymal stem cell (ADMSC) therapy can promote recovery from cerebral ischemia; however, more information regarding appropriate sources of ADMSCs is required. This study was aimed at analyzi...

    Authors: Zhang Yu, Tang Wenyan, Su Xuewen, Dong Baixiang, Wang Qian, Wang Zhaoyan, Yang Yinxiang, Qu Suqing and Luan Zuo
    Citation: Journal of Translational Medicine 2018 16:339
    Content type: Research Published on:

  39. At present, many laboratories and hospitals all over the world are attempting and exploring the clinical transformation of this tissue engineered bone graft (TEBG) strategy. Many successful cases of bone tissu...

    Authors: Dingyu Wu, Zhenxing Wang, Zhiwei Zheng, Yingnan Geng, Zhanzhao Zhang, Qiannan Li, Quan Zhou, Yilin Cao and Zhi-Yong Zhang
    Citation: Journal of Translational Medicine 2018 16:324
    Content type: Research Published on:

  40. Bone is a highly vascularized and resilient organ with innate healing abilities, however some bone injuries overwhelm these attributes and require intervention, such as bone tissue engineering strategies. Comb...

    Authors: Emily L. Durham, R. Nicole Howie, SarahRose Hall, Nicholas Larson, Brayden Oakes, Reed Houck, Zachary Grey, Martin Steed, Amanda C. LaRue, Robin Muise-Helmericks and James Cray
    Citation: Journal of Translational Medicine 2018 16:321
    Content type: Research Published on:

  41. Successful delivery of cell-based therapeutics into patients is compromised by their short shelf-life upon release from production facilities due to the living nature of the active component that rapidly loses...

    Authors: Clémentine Mirabel, Eduard Puente-Massaguer, Anna del Mazo-Barbara, Blanca Reyes, Philip Morton, Francesc Gòdia and Joaquim Vives
    Citation: Journal of Translational Medicine 2018 16:291
    Content type: Methodology Published on:

  42. Cardiomyocytes derived from animals and induced pluripotent stem cells (iPSCs) are two main cellular models to study cardiovascular diseases, however, neither provides precise modeling of the response of matur...

    Authors: Guang-ran Guo, Liang Chen, Man Rao, Kai Chen, Jiang-ping Song and Sheng-shou Hu
    Citation: Journal of Translational Medicine 2018 16:288
    Content type: Methodology Published on:

  43. New technologies and therapies designed to facilitate development of personalized treatments are rapidly emerging in the field of biomedicine. Strikingly, the goal of personalized medicine refined the concept ...

    Authors: Sara Deola, Bella S. Guerrouahen, Heba Sidahmed, Anjud Al-Mohannadi, Muhammad Elnaggar, Ramaz Elsadig, Essam M. Abdelalim, Goran Petrovski, Massimo Gadina, Adrian Thrasher, Winfried S. Wels, Stephen P. Hunger, Ena Wang, Francesco M. Marincola, Cristina Maccalli and Chiara Cugno
    Citation: Journal of Translational Medicine 2018 16:276
    Content type: Meeting report Published on:

  44. Bone marrow concentrate (BMC) has shown promise in the treatment of several orthopedic conditions. This registry study investigated the use of autologous BMC and platelet products for percutaneous anterior cru...

    Authors: Christopher Centeno, Jason Markle, Ehren Dodson, Ian Stemper, Christopher Williams, Matthew Hyzy, Thomas Ichim and Michael Freeman
    Citation: Journal of Translational Medicine 2018 16:246
    Content type: Research Published on:

  45. Tumor necrosis factor (TNF)-alpha was originally identified in the 1970s as the serum mediator of innate immunity capable of inducing hemorrhagic necrosis in tumors. Today, a wide spectrum of biological activi...

    Authors: Steven F. Josephs, Thomas E. Ichim, Stephen M. Prince, Santosh Kesari, Francesco M. Marincola, Anton Rolando Escobedo and Amir Jafri
    Citation: Journal of Translational Medicine 2018 16:242
    Content type: Review Published on:

  46. Mesenchymal stem cells (MSCs) represent an attractive avenue for cellular therapies targeting degenerative diseases. MSC in vitro expansion is required in order to obtain therapeutic numbers during the manufac...

    Authors: C. Elabd, T. E. Ichim, K. Miller, A. Anneling, V. Grinstein, V. Vargas and F. J. Silva
    Citation: Journal of Translational Medicine 2018 16:222
    Content type: Research Published on:

  47. Mesenchymal stromal cells (MSCs) are a promising option to treat knee osteoarthritis (OA). Their safety and usefulness have been reported in several short-term clinical trials but less information is available...

    Authors: José María Lamo-Espinosa, Gonzalo Mora, Juan F. Blanco, Froilán Granero-Moltó, Jorge María Núñez-Córdoba, Silvia López-Elío, Enrique Andreu, Fermín Sánchez-Guijo, José Dámaso Aquerreta, José María Bondía, Andrés Valentí-Azcárate, María del Consuelo del Cañizo, Eva María Villarón, Juan Ramón Valentí-Nin and Felipe Prósper
    Citation: Journal of Translational Medicine 2018 16:213
    Content type: Commentary Published on:

  48. Mesenchymal stem cell (MSC) therapy offers great potential for treatment of disease through the multifunctional and responsive ability of these cells. In numerous contexts, MSC have been shown to reduce inflam...

    Authors: Thomas E. Ichim, Pete O’Heeron and Santosh Kesari
    Citation: Journal of Translational Medicine 2018 16:212
    Content type: Review Published on:

  49. Necroptotic susceptibility is probably an intrinsic weakness of cancer. Here, we report that resibufogenin, a member of bufadienolide family, suppresses the growth and metastasis of colorectal cancer (CRC) thr...

    Authors: Qinrui Han, Ye Ma, Hao Wang, Yu Dai, Chunhui Chen, Yawei Liu, Linlin Jing and Xuegang Sun
    Citation: Journal of Translational Medicine 2018 16:201
    Content type: Research Published on:

  50. Stored platelets undergo deleterious changes, referred to as platelet storage lesions (PSLs), which accelerate the desialylation of platelets and result in their phagocytosis and clearance by hepatic macrophag...

    Authors: Jooyoung Cho, Hyunkyung Kim, Jaewoo Song, June-Won Cheong, Jeong Won Shin, Woo Ick Yang and Hyun Ok Kim
    Citation: Journal of Translational Medicine 2018 16:199
    Content type: Research Published on:
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