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Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
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References

Aiuti, A., F. Cattaneo, S. Galimberti, U. Benninghoff, B. Cassani, L. Callegaro, S. Scaramuzza, G. Andolfi, M. Mirolo, I. Brigida, A. Tabucchi, F. Carlucci, M. Eibl, M. Aker, S. Slavin, H. Al-Mousa, A. Al Ghonaium, A. Ferster, A. Duppenthaler, L. Notarangelo, U. Wintergerst, R. H. Buckley, M. Bregni, S. Marktel, M. G. Valsecchi, P. Rossi, F. Ciceri, R. Miniero, C. Bordignon, and M. G. Roncarolo. 2009. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. New England Journal of Medicine 360(5):447–458.

Al-Awqati, Q., and J. A. Oliver. 2002. Stem cells in the kidney. Kidney International 61(2):387–395.

Aminzadeh, M. A., E. Tseliou, B. Sun, K. Cheng, K. Malliaras, R. R. Makkar, and E. Marban. 2015. Therapeutic efficacy of cardiosphere-derived cells in a transgenic mouse model of non-ischaemic dilated cardiomyopathy. European Heart Journal 36(12):751–762.

AOA (American Optometric Association). 1994. Care of the patient with age-related macular degeneration. http://www.aoa.org/documents/optometrists/QRG-6.pdf (accessed December 12, 2016).

ASHSP (American Society of Health-System Pharmacists). 2016. Tolvaptan. https://medlineplus.gov/druginfo/meds/a609033.html (accessed December 12, 2016).

Awad, A. S., L. Huang, H. Ye, E. T. Duong, W. K. Bolton, J. Linden, and M. D. Okusa. 2006. Adenosine a2a receptor activation attenuates inflammation and injury in diabetic nephropathy. American Journal of Physiology—Renal Physiology 290(4):F828–F837.

Benam, K. H., R. Villenave, C. Lucchesi, A. Varone, C. Hubeau, H. H. Lee, S. E. Alves, M. Salmon, T. C. Ferrante, J. C. Weaver, A. Bahinski, G. A. Hamilton, and D. E. Ingber. 2016. Small airway-on-a-chip enables analysis of human lung inflammation and drug responses in vitro. Nature Methods 13(2):151–157.

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
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Braun, C. J., K. Boztug, A. Paruzynski, M. Witzel, A. Schwarzer, M. Rothe, U. Modlich, R. Beier, G. Gohring, D. Steinemann, R. Fronza, C. R. Ball, R. Haemmerle, S. Naundorf, K. Kuhlcke, M. Rose, C. Fraser, L. Mathias, R. Ferrari, M. R. Abboud, W. Al-Herz, I. Kondratenko, L. Marodi, H. Glimm, B. Schlegelberger, A. Schambach, M. H. Albert, M. Schmidt, C. von Kalle, and C. Klein. 2014. Gene therapy for Wiskott–Aldrich syndrome—Long-term efficacy and genotoxicity. Science Translational Medicine 6(227):227ra233.

Brentjens, R. J., J. B. Latouche, E. Santos, F. Marti, M. C. Gong, C. Lyddane, P. D. King, S. Larson, M. Weiss, I. Riviere, and M. Sadelain. 2003. Eradication of systemic B cell tumors by genetically targeted human T lymphocytes co-stimulated by CD80 and interleukin-15. Nature Medicine 9(3):279–286.

Business Wire. 2013. Otsuka receives complete response letter from U.S. Food and Drug Administration for tolvaptan for use in patients with autosomal dominant polycystic kidney disease. Business Wire, August 29. http://www.businesswire.com/news/home/20130829006215/en/Otsuka-Receives-Complete-Response-Letter-U.S.-Food (accessed January 3, 2017).

Cartier, N., S. Hacein-Bey-Abina, C. C. Bartholomae, G. Veres, M. Schmidt, I. Kutschera, M. Vidaud, U. Abel, L. Dal-Cortivo, L. Caccavelli, N. Mahlaoui, V. Kiermer, D. Mittelstaedt, C. Bellesme, N. Lahlou, F. Lefrere, S. Blanche, M. Audit, E. Payen, P. Leboulch, B. l'Homme, P. Bougneres, C. Von Kalle, A. Fischer, M. Cavazzana-Calvo, and P. Aubourg. 2009. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326(5954):818–823.

Cavazzana-Calvo, M., E. Payen, O. Negre, G. Wang, K. Hehir, F. Fusil, J. Down, M. Denaro, T. Brady, K. Westerman, R. Cavallesco, B. Gillet-Legrand, L. Caccavelli, R. Sgarra, L. Maouche-Chretien, F. Bernaudin, R. Girot, R. Dorazio, G. J. Mulder, A. Polack, A. Bank, J. Soulier, J. Larghero, N. Kabbara, B. Dalle, B. Gourmel, G. Socie, S. Chretien, N. Cartier, P. Aubourg, A. Fischer, K. Cornetta, F. Galacteros, Y. Beuzard, E. Gluckman, F. Bushman, S. Hacein-Bey-Abina, and P. Leboulch. 2010. Transfusion independence and HMGA2 activation after gene therapy of human betathalassaemia. Nature 467(7313):318–322.

CDC (Centers for Disease Control and Prevention). 2016. Heart failure fact sheet. http://www.cdc.gov/dhdsp/data_statistics/fact_sheets/fs_heart_failure.htm (accessed December 1, 2016).

Chhabra, A., A. M. Ring, K. Weiskopf, P. J. Schnorr, S. Gordon, A. C. Le, H. S. Kwon, N. G. Ring, J. Volkmer, P. Y. Ho, S. Tseng, I. L. Weissman, and J. A. Shizuru. 2016. Hematopoietic stem cell transplantation in immunocompetent hosts without radiation or chemotherapy. Science Translational Medicine 8(351):351ra105.

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

Chimenti, I., R. R. Smith, T. S. Li, G. Gerstenblith, E. Messina, A. Giacomello, and E. Marban. 2010. Relative roles of direct regeneration versus paracrine effects of human cardiosphere-derived cells transplanted into infarcted mice. Circulation Research 106(5):971–980.

Cummings, B. J., N. Uchida, S. J. Tamaki, D. L. Salazar, M. Hooshmand, R. Summers, F. H. Gage, and A. J. Anderson. 2005. Human neural stem cells differentiate and promote locomotor recovery in spinal cord-injured mice. Proceedings of the National Academy of Sciences of the United States of America 102(39):14069–14074.

De Ravin, S. S., X. Wu, S. Moir, S. Anaya-O’Brien, N. Kwatemaa, P. Littel, N. Theobald, U. Choi, L. Su, M. Marquesen, D. Hilligoss, J. Lee, C. M. Buckner, K. A. Zarember, G. O’Connor, D. McVicar, D. Kuhns, R. E. Throm, S. Zhou, L. D. Notarangelo, I. C. Hanson, M. J. Cowan, E. Kang, C. Hadigan, M. Meagher, J. T. Gray, B. P. Sorrentino, and H. L. Malech. 2016. Lentiviral hematopoietic stem cell gene therapy for x-linked severe combined immunodeficiency. Science Translational Medicine (335):335ra357.

Doudna, J. A., and E. Charpentier. 2014. Genome editing. The new frontier of genome engineering with CRISPR-Cas9. Science 346(6213):1258096.

Feldman, E. L., N. M. Boulis, J. Hur, K. Johe, S. B. Rutkove, T. Federici, M. Polak, J. Bordeau, S. A. Sakowski, and J. D. Glass. 2014. Intraspinal neural stem cell transplantation in amyotrophic lateral sclerosis: Phase 1 trial outcomes. Annals of Neurology 75(3):363–373.

Fire, A., S. Xu, M. K. Montgomery, S. A. Kostas, S. E. Driver, and C. C. Mello. 1998. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391(6669):806–811.

Freeman, A. F., N. N. Shah, M. Parta, H. C. Su, A. Brofferio, I. Gradus-Pizlo, S. Butty, T. E. Hughes, D. E. Kleiner, D. Avila, T. Heller, H. H. Kong, S. M. Holland, and D. D. Hickstein. 2016. Haploidentical related donor hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for DOCK8 deficiency. Journal of Allergy and Clinical Immunology: In Practice 4(6):1239–1242, e1231.

Fu, J. D., N. R. Stone, L. Liu, C. I. Spencer, L. Qian, Y. Hayashi, P. Delgado-Olguin, S. Ding, B. G. Bruneau, and D. Srivastava. 2013. Direct reprogramming of human fibroblasts toward a cardiomyocyte-like state. Stem Cell Reports 1(3):235–247.

Gaspar, H. B., S. Cooray, K. C. Gilmour, K. L. Parsley, F. Zhang, S. Adams, E. Bjorkegren, J. Bayford, L. Brown, E. G. Davies, P. Veys, L. Fairbanks, V. Bordon, T. Petropoulou, C. Kinnon, and A. J. Thrasher. 2011. Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Science Translational Medicine 3(97):97ra80.

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

Grantham, J. J., V. E. Torres, A. B. Chapman, L. M. Guay-Woodford, K. T. Bae, B. F. J. King, L. H. Wetzel, D. A. Baumgarten, P. J. Kenney, P. C. Harris, S. Klahr, W. M. Bennett, G. N. Hirschman, C. M. Meyers, X. Zhang, F. Zhu, and J. P. Miller. 2006. Volume progression in polycystic kidney disease. New England Journal of Medicine 354(20):2122–2130.

Griffiths, A., W. Gelbart, J. H. Miller, and R. C. Lewontin. 1999. Making recombinant DNA. Chapter 10, section 2 in Modern genetic analysis. New York: W. H. Freeman.

Hacein-Bey-Abina, S., A. Garrigue, G. P. Wang, J. Soulier, A. Lim, E. Morillon, E. Clappier, L. Caccavelli, E. Delabesse, K. Beldjord, V. Asnafi, E. MacIntyre, L. Dal Cortivo, I. Radford, N. Brousse, F. Sigaux, D. Moshous, J. Hauer, A. Borkhardt, B. H. Belohradsky, U. Wintergerst, M. C. Velez, L. Leiva, R. Sorensen, N. Wulffraat, S. Blanche, F. D. Bushman, A. Fischer, and M. Cavazzana-Calvo. 2008. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. Journal of Clinical Investigation 118(9):3132–3142.

Hacein-Bey-Abina, S., J. Hauer, A. Lim, C. Picard, G. P. Wang, C. C. Berry, C. Martinache, F. Rieux-Laucat, S. Latour, B. H. Belohradsky, L. Leiva, R. Sorensen, M. Debre, J. L. Casanova, S. Blanche, A. Durandy, F. D. Bushman, A. Fischer, and M. Cavazzana-Calvo. 2010. Efficacy of gene therapy for X-linked severe combined immunodeficiency. New England Journal of Medicine 363(4):355–364.

Hacein-Bey-Abina, S., S. Y. Pai, H. B. Gaspar, M. Armant, C. C. Berry, S. Blanche, J. Bleesing, J. Blondeau, H. de Boer, K. F. Buckland, L. Caccavelli, G. Cros, S. De Oliveira, K. S. Fernandez, D. Guo, C. E. Harris, G. Hopkins, L. E. Lehmann, A. Lim, W. B. London, J. C. van der Loo, N. Malani, F. Male, P. Malik, M. A. Marinovic, A. M. McNicol, D. Moshous, B. Neven, M. Oleastro, C. Picard, J. Ritz, C. Rivat, A. Schambach, K. L. Shaw, E. A. Sherman, L. E. Silberstein, E. Six, F. Touzot, A. Tsytsykova, J. Xu-Bayford, C. Baum, F. D. Bushman, A. Fischer, D. B. Kohn, A. H. Filipovich, L. D. Notarangelo, M. Cavazzana, D. A. Williams, and A. J. Thrasher. 2014. A modified gamma-retrovirus vector for X-linked severe combined immunodeficiency. New England Journal of Medicine 371(15):1407–1417.

Hacein-Bey-Abina, S., H. B. Gaspar, J. Blondeau, L. Caccavelli, S. Charrier, K. Buckland, C. Picard, E. Six, N. Himoudi, K. Gilmour, A. M. McNicol, H. Hara, J. Xu-Bayford, C. Rivat, F. Touzot, F. Mavilio, A. Lim, J. M. Treluyer, S. Heritier, F. Lefrere, J. Magalon, I. Pengue-Koyi, G. Honnet, S. Blanche, E. A. Sherman, F. Male, C. Berry, N. Malani, F. D. Bushman, A. Fischer, A. J. Thrasher, A. Galy, and M. Cavazzana. 2015. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA 313(15):1550–1563.

Holt, N., J. Wang, K. Kim, G. Friedman, X. Wang, V. Taupin, G. M. Crooks, D. B. Kohn, P. D. Gregory, M. C. Holmes, and P. M. Cannon. 2010. Human

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nature Biotechnology 28(8):839–847.

Howe, S. J., M. R. Mansour, K. Schwarzwaelder, C. Bartholomae, M. Hubank, H. Kempski, M. H. Brugman, K. Pike-Overzet, S. J. Chatters, D. de Ridder, K. C. Gilmour, S. Adams, S. I. Thornhill, K. L. Parsley, F. J. Staal, R. E. Gale, D. C. Linch, J. Bayford, L. Brown, M. Quaye, C. Kinnon, P. Ancliff, D. K. Webb, M. Schmidt, C. von Kalle, H. B. Gaspar, and A. J. Thrasher. 2008. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. Journal of Clinical Investigation 118(9):3143–3150.

Ibrahim, A. G., K. Cheng, and E. Marban. 2014. Exosomes as critical agents of cardiac regeneration triggered by cell therapy. Stem Cell Reports 2(5):606–619.

Ieda, M., J. D. Fu, P. Delgado-Olguin, V. Vedantham, Y. Hayashi, B. G. Bruneau, and D. Srivastava. 2010. Direct reprogramming of fibroblasts into functional cardiomyocytes by defined factors. Cell 142(3):375–386.

Karpati, G., D. Ajdukovic, D. Arnold, R. B. Gledhill, R. Guttmann, P. Holland, P. A. Koch, E. Shoubridge, D. Spence, M. Vanasse, et al. 1993. Myoblast transfer in Duchenne muscular dystrophy. Annals of Neurology 34(1):8–17.

Kemter, E., and E. Wolf. 2015. Pigs pave a way to de novo formation of functional human kidneys. Proceedings of the National Academy of Sciences of the United States of America 112(42):12905–12906.

Klug, A. 2010. The discovery of zinc fingers and their development for practical applications in gene regulation and genome manipulation. Quarterly Reviews of Biophysics 43(1):1–21.

Kohler, G., and C. Milstein. 1975. Continuous cultures of fused cells secreting antibody of predefined specificity. Nature 256(5517):495–497.

Lancaster, M. A., and J. A. Knoblich. 2014. Organogenesis in a dish: Modeling development and disease using organoid technologies. Science 345(6194):232–292.

Lappas, C. M., Y. J. Day, M. A. Marshall, V. H. Engelhard, and J. Linden. 2006. Adenosine A2A receptor activation reduces hepatic ischemia reperfusion injury by inhibiting CD1d-dependent NKT cell activation. Journal of Experimental Medicine 203(12):2639–2648.

Lappas, C. M., P. C. Liu, J. Linden, E. M. Kang, and H. L. Malech. 2010. Adenosine A2A receptor activation limits graft-versus-host disease after allogeneic hematopoietic stem cell transplantation. Journal of Leukocyte Biology 87(2):345–354.

Le Tissier, P., J. P. Stoye, Y. Takeuchi, C. Patience, and R. A. Weiss. 1997. Two sets of human-tropic pig retrovirus. Nature 389(6652):681–682.

Leen, A. M., C. M. Bollard, A. M. Mendizabal, E. J. Shpall, P. Szabolcs, J. H. Antin, N. Kapoor, S. Y. Pai, S. D. Rowley, P. Kebriaei, B. R. Dey, B. J. Grilley, A. P. Gee, M. K. Brenner, C. M. Rooney, and H. E. Heslop. 2013. Multicenter study of banked third-party virus-specific T cells to treat severe

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

viral infections after hematopoietic stem cell transplantation. Blood 121(26):5113–5123.

Leen, A. M., H. E. Heslop, and M. K. Brenner. 2014. Antiviral T cell therapy. Immunological Reviews 258(1):12–29.

Leukemia & Lymphoma Society. 2016. Graft versus host disease. https://www.lls.org/treatment/types-of-treatment/stem-cell-transplantation/graft-versus-host-disease (accessed January 3, 2017).

Li, T. S., K. Cheng, K. Malliaras, R. R. Smith, Y. Zhang, B. Sun, N. Matsushita, A. Blusztajn, J. Terrovitis, H. Kusuoka, L. Marban, and E. Marban. 2012. Direct comparison of different stem cell types and subpopulations reveals superior paracrine potency and myocardial repair efficacy with cardiosphere-derived cells. Journal of the American College of Cardiology 59(10):942–953.

Liu, R., W. A. Paxton, S. Choe, D. Ceradini, S. R. Martin, R. Horuk, M. E. MacDonald, H. Stuhlmann, R. A. Koup, and N. R. Landau. 1996. Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection. Cell 86(3):367–377.

Locatelli, F., D. Pende, M. C. Mingari, A. Bertaina, M. Falco, A. Moretta, and L. Moretta. 2013. Cellular and molecular basis of haploidentical hematopoietic stem cell transplantation in the successful treatment of high-risk leukemias: Role of alloreactive NK cells. Frontiers in Immunology 4(15).

Maffioletti, S. M., M. Noviello, K. English, and F. S. Tedesco. 2014. Stem cell transplantation for muscular dystrophy: The challenge of immune response. BioMed Research International 2014.

Makkar, R. R., R. R. Smith, K. Cheng, K. Malliaras, L. E. Thomson, D. Berman, L. S. Czer, L. Marban, A. Mendizabal, P. V. Johnston, S. D. Russell, K. H. Schuleri, A. C. Lardo, G. Gerstenblith, and E. Marban. 2012. Intracoronary cardiosphere-derived cells for heart regeneration after myocardial infarction (CADUCEUS): A prospective, randomised phase 1 trial. Lancet 379(9819):895–904.

Malech, H. L., and H. D. Ochs. 2015. An emerging era of clinical benefit from gene therapy. JAMA 313(15):1522–1523.

Mohamed, T. M., N. R. Stone, E. C. Berry, E. Radzinsky, Y. Huang, K. Pratt, Y. S. Ang, P. Yu, H. Wang, S. Tang, S. Magnitsky, S. Ding, K. N. Ivey, and D. Srivastava. 2017. Chemical enhancement of in vitro and in vivo direct cardiac reprogramming. Circulation 135(10):978–995.

Naganuma, M., E. B. Wiznerowicz, C. M. Lappas, J. Linden, M. T. Worthington, and P. B. Ernst. 2006. Cutting edge: Critical role for A2A adenosine receptors in the T cell–mediated regulation of colitis. Journal of Immunology 177(5):2765–2769.

Nagashima, H., and H. Matsunari. 2016. Growing human organs in pigs—A dream or reality? Theriogenology 86(1):422–426.

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

National Kidney Foundation. 2015. Dialysis. https://www.kidney.org/atoz/content/dialysisinfo (accessed December 12, 2016).

———. 2016a. End stage renal disease in the United States. https://www/kidney.org/news/newsroom/factsheets/End-Stage-Renal-Disease-in-the-US (accessed December 12, 2016).

———. 2016b. Fast facts. https://www.kidney.org/news/newsroom/factsheets/FastFacts (accessed December 12, 2016).

———. 2016c. Polycystic kidney disease. https://www.kidney.org/atoz/content/polycystic (accessed December 12, 2016).

NIDDK (National Institute of Diabetes and Digestive and Kidney Diseases). 2015. Polycystic kidney disease. https://www.niddk.nih.gov/health-information/health-topics/kidney-disease/polycystic-kidney-disease-pkd/Pages/facts.aspx#eating (accessed December 12, 2016).

Olsan, E. E., T. Matsushita, M. Rezaei, and T. Weimbs. 2015. Exploitation of the polymeric immunoglobulin receptor for antibody targeting to renal cyst lumens in polycystic kidney disease. Journal of Biological Chemistry 290(25):15679–15686.

Palchaudhuri, R., B. Saez, J. Hoggatt, A. Schajnovitz, D. B. Sykes, T. A. Tate, A. Czechowicz, Y. Kfoury, F. Ruchika, D. J. Rossi, G. L. Verdine, M. K. Mansour, and D. T. Scadden. 2016. Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin. Nature Biotechnology 34(7):738–745.

Parta, M., D. Hilligoss, C. Kelly, N. Kwatemaa, N. Theobald, H. Malech, and E. M. Kang. 2015. Haploidentical hematopoietic cell transplantation with post-transplant cyclophosphamide in a patient with chronic granulomatous disease and active infection: A first report. Journal of Clinical Immunology 35(7):675–680.

Patience, C., Y. Takeuchi, and R. A. Weiss. 1997. Infection of human cells by an endogenous retrovirus of pigs. Nature Medicine 3(3):282–286.

PKD (Polycystic Kidney Disease) Foundation. 2013. FDA responds to tolvaptan new drug application. https://pkdcure.org/2013/08/29/fda-responds-tolvaptan-new-drug-application (accessed December 12, 2016).

———. 2016. What is PKD? https://pkdcure.org/what-is-pkd (accessed December 12, 2016).

Qian, L., Y. Huang, C. I. Spencer, A. Foley, V. Vedantham, L. Liu, S. J. Conway, J. D. Fu, and D. Srivastava. 2012. In vivo reprogramming of murine cardiac fibroblasts into induced cardiomyocytes. Nature 485(7400):593–598.

Ratcliffe, A. 2004. The clinical market for tissue engineered products in orthopaedics. In Tissue engineering in musculoskeletal clinical practice, edited by L. Sandell and A. Grodzinsky. Rosemont, IL: American Academy of Orthopaedic Surgeons. P. 17.

Sadelain, M. 2015. CAR therapy: The CD19 paradigm. Journal of Clinical Investigation 125(9):3392–3400.

Scullideri, M. 2016. How iPS cells changed the world. Nature 534(7607):310–312.

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

Sebastiano, V., H. H. Zhen, B. Haddad, E. Bashkirova, S. P. Melo, P. Wang, T. L. Leung, Z. Siprashvili, A. Tichy, J. Li, M. Ameen, J. Hawkins, S. Lee, L. Li, A. Schwertschkow, G. Bauer, L. Lisowski, M. A. Kay, S. K. Kim, A. T. Lane, M. Wernig, and A. E. Oro. 2014. Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa. Science Translational Medicine 6(264):264ra163.

Sessa, M., L. Lorioli, F. Fumagalli, S. Acquati, D. Redaelli, C. Baldoli, S. Canale, I. D. Lopez, F. Morena, A. Calabria, R. Fiori, P. Silvani, P. M. Rancoita, M. Gabaldo, F. Benedicenti, G. Antonioli, A. Assanelli, M. P. Cicalese, U. Del Carro, M. G. Sora, S. Martino, A. Quattrini, E. Montini, C. Di Serio, F. Ciceri, M. G. Roncarolo, A. Aiuti, L. Naldini, and A. Biffi. 2016. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: An ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet 388(10043):476–487.

Shapiro, A. M. J., J. R. T. Lakey, E. A. Ryan, G. S. Korbutt, E. Toth, G. L. Warnock, N. M. Kneteman, and R. V. Rajotte. 2000. Islet transplantation in seven patients with type 1 diabetes mellitus using a glucocorticoid-free immunosuppressive regimen. New England Journal of Medicine 343(4): 230–238.

Shiba, Y., T. Gomibuchi, T. Seto, Y. Wada, H. Ichimura, Y. Tanaka, T. Ogasawara, K. Okada, N. Shiba, K. Sakamoto, D. Ido, T. Shiina, M. Ohkura, J. Nakai, N. Uno, Y. Kazuki, M. Oshimura, I. Minami, and U. Ikeda. 2016. Allogeneic transplantation of iPS cell–derived cardiomyocytes regenerates primate hearts. Nature 538(7625):388–391.

Shillingford, J. M., C. P. Leamon, I. R. Vlahov, and T. Weimbs. 2012. Folateconjugated rapamycin slows progression of polycystic kidney disease. Journal of the American Society of Nephrology 23(10):1674–1681.

Siprashvili, Z., N. T. Nguyen, E. S. Gorell, K. Loutit, P. Khuu, L. K. Furukawa, H. P. Lorenz, T. H. Leung, D. R. Keene, K. E. Rieger, P. Khavari, A. T. Lane, J. Y. Tang, and M. P. Marinkovich. 2016. Safety and wound outcomes following genetically corrected autologous epidermal grafts in patients with recessive dystrophic epidermolysis bullosa. JAMA 316(17):1808–1817.

Smith, R. R., L. Barile, H. C. Cho, M. K. Leppo, J. M. Hare, E. Messina, A. Giacomello, M. R. Abraham, and E. Marban. 2007. Regenerative potential of cardiosphere-derived cells expanded from percutaneous endomyocardial biopsy specimens. Circulation 115(7):896–908.

Sonoda, S., P. G. Sreekumar, S. Kase, C. Spee, S. J. Ryan, R. Kannan, and D. R. Hinton. 2009. Attainment of polarity promotes growth factor secretion by retinal pigment epithelial cells: Relevance to age-related macular degeneration. Aging (Albany, NY) 2(1):28–42.

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

Stein, S., M. G. Ott, S. Schultze-Strasser, A. Jauch, B. Burwinkel, A. Kinner, M. Schmidt, A. Kramer, J. Schwable, H. Glimm, U. Koehl, C. Preiss, C. Ball, H. Martin, G. Gohring, K. Schwarzwaelder, W. K. Hofmann, K. Karakaya, S. Tchatchou, R. Yang, P. Reinecke, K. Kuhlcke, B. Schlegelberger, A. J. Thrasher, D. Hoelzer, R. Seger, C. von Kalle, and M. Grez. 2010. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nature Medicine 16(2):198–204.

Takasato, M., P. X. Er, H. S. Chiu, B. Maier, G. J. Baillie, C. Ferguson, R. G. Parton, E. J. Wolvetang, M. S. Roost, S. M. Chuva de Sousa Lopes, and M. H. Little. 2015. Kidney organoids from human iPS cells contain multiple lineages and model human nephrogenesis. Nature 526(7574):564–568.

Tamaki, S. J., Y. Jacobs, M. Dohse, A. Capela, J. D. Cooper, M. Reitsma, D. He, R. Tushinski, P. V. Belichenko, A. Salehi, W. Mobley, F. H. Gage, S. Huhn, A. S. Tsukamoto, I. L. Weissman, and N. Uchida. 2009. Neuroprotection of host cells by human central nervous system stem cells in a mouse model of infantile neuronal ceroid lipofuscinosis. Cell Stem Cell 5(3):310–319.

Tebas, P., D. Stein, W. W. Tang, I. Frank, S. Q. Wang, G. Lee, S. K. Spratt, R. T. Surosky, M. A. Giedlin, G. Nichol, M. C. Holmes, P. D. Gregory, D. G. Ando, M. Kalos, R. G. Collman, G. Binder-Scholl, G. Plesa, W. T. Hwang, B. L. Levine, and C. H. June. 2014. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. New England Journal of Medicine 370(10):901–910.

Themeli, M., C. C. Kloss, G. Ciriello, V. D. Fedorov, F. Perna, M. Gonen, and M. Sadelain. 2013. Generation of tumor-targeted human T lymphocytes from induced pluripotent stem cells for cancer therapy. Nature Biotechnology 31(10):928–933.

Trounson, A., and C. McDonald. 2015. Stem cell therapies in clinical trials: Progress and challenges. Cell Stem Cell 17(1):11–22.

Tseliou, E., G. de Couto, J. Terrovitis, B. Sun, L. Weixin, L. Marban, and E. Marban. 2014a. Angiogenesis, cardiomyocyte proliferation and anti-fibrotic effects underlie structural preservation post-infarction by intramyocardiallyinjected cardiospheres. PLOS ONE 9(2):e88590.

Tseliou, E., H. Reich, G. de Couto, J. Terrovitis, B. Sun, W. Liu, and E. Marban. 2014b. Cardiospheres reverse adverse remodeling in chronic rat myocardial infarction: Roles of soluble endoglin and Tgf-beta signaling. Basic Research in Cardiology 109(6):443.

Tsukamoto, A., S. E. Abbot, L. C. Kadyk, N. D. DeWitt, D. V. Schaffer, J. A. Wertheim, K. J. Whittlesey, and M. J. Werner. 2016. Challenging regeneration to transform medicine. Stem Cells Translational Medicine 5(1):1–7.

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×

Uchida, N., K. Chen, M. Dohse, K. D. Hansen, J. Dean, J. R. Buser, A. Riddle, D. J. Beardsley, Y. Wan, X. Gong, T. Nguyen, B. J. Cummings, A. J. Anderson, S. J. Tamaki, A. Tsukamoto, I. L. Weissman, S. G. Matsumoto, L. S. Sherman, C. D. Kroenke, and S. A. Back. 2012. Human neural stem cells induce functional myelination in mice with severe dysmyelination. Science Translational Medicine 4(155):155ra136.

U.S. Renal Data System. 2013. Costs of ESRD. https://www.usrds.org/2013/view/v2_11.aspx (accessed December 12, 2016).

van der Laan, L. J., C. Lockey, B. C. Griffeth, F. S. Frasier, C. A. Wilson, D. E. Onions, B. J. Hering, Z. Long, E. Otto, B. E. Torbett, and D. R. Salomon. 2000. Infection by porcine endogenous retrovirus after islet xenotransplantation in SCID mice. Nature 407(6800):90–94.

Wang, K., G. Wei, and D. Liu. 2012. CD19: A biomarker for B cell development, lymphoma diagnosis and therapy. Experimental Hematology & Oncology 1(1):36.

Wilson, C. A. 2008. Porcine endogenous retroviruses and xenotransplantation. Cellular and Molecular Life Sciences 65(21):3399–3412.

Wu, L., N. P. Gerard, R. Wyatt, H. Choe, C. Parolin, N. Ruffing, A. Borsetti, A. A. Cardoso, E. Desjardin, W. Newman, C. Gerard, and J. Sodroski. 1996. CD4-induced interaction of primary HIV-1 gp120 glycoproteins with the chemokine receptor CCR-5. Nature 384(6605):179–183.

Yang, L., M. Guell, D. Niu, H. George, E. Lesha, D. Grishin, J. Aach, E. Shrock, W. Xu, J. Poci, R. Cortazio, R. A. Wilkinson, J. A. Fishman, and G. Church. 2015. Genome-wide inactivation of porcine endogenous retroviruses (PERVS). Science 350(6264):1101–1104.

Zarek, P. E., C. T. Huang, E. R. Lutz, J. Kowalski, M. R. Horton, J. Linden, C. G. Drake, and J. D. Powell. 2008. A2A receptor signaling promotes peripheral tolerance by inducing T cell anergy and the generation of adaptive regulatory T cells. Blood 111(1):251–259.

Zou, S., S. Glynn, D. Kuritzkes, M. Shah, N. Cook, N. Berliner, and NHLBI AIDS Blood Session Working Group. 2013. Hematopoietic cell transplantation and HIV cure: Where we are and what next? Blood 122(18):3111–3115.

Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
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Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 78
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 79
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 80
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 81
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 82
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 83
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 84
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 85
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24671.
×
Page 86
Next: Appendix A: Workshop Agenda »
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