
ISSCR Continuing Education Course
References & Resources
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References:
Blau HM, Daley GQ. Stem Cells in the Treatment of Disease [published correction appears in N Engl J Med. 2019 Aug 29;381(9):890. doi: 10.1056/NEJMx190025.]. N Engl J Med. 2019;380(18):1748-1760. doi:10.1056/NEJMra1716145
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References:
Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. N Engl J Med. 2021;384(3):252-260. doi:10.1056/NEJMoa2031054
Gallico GG 3rd, O'Connor NE, Compton CC, Kehinde O, Green H. Permanent coverage of large burn wounds with autologous cultured human epithelium. N Engl J Med. 1984;311(7):448-451. doi:10.1056/NEJM198408163110706
Hirsch T, Rothoeft T, Teig N, et al. Regeneration of the entire human epidermis using transgenic stem cells. Nature. 2017;551(7680):327-332. doi:10.1038/nature24487
Kim et al. Biphasic Activation of WNT Signaling Facilitates the Derivation of Midbrain Dopamine Neurons from hESCs for Translational Use. Cell Stem Cell 2021 Feb 4;28(2):343-355.e5. doi: 10.1016/j.stem.2021.01.005.
Kirkeby A, Main H, Carpenter M. Pluripotent stem-cell-derived therapies in clinical trial: A 2025 update [published correction appears in Cell Stem Cell. 2025 Feb 6;32(2):329-331. doi: 10.1016/j.stem.2025.01.003.]. Cell Stem Cell. 2025;32(1):10-37. doi:10.1016/j.stem.2024.12.005
Melenhorst JJ, Chen GM, Wang M, et al. Decade-long leukaemia remissions with persistence of CD4+ CAR T cells [published correction appears in Nature. 2022 Dec;612(7941):E22. doi: 10.1038/s41586-022-05376-8.]. Nature. 2022;602(7897):503-509. doi:10.1038/s41586-021-04390-6
Pagliuca FW, Millman JR, Gürtler M, et al. Generation of functional human pancreatic β cells in vitro. Cell. 2014;159(2):428-439. doi:10.1016/j.cell.2014.09.040
Piao et al. Preclinical Efficacy and Safety of a Human Embryonic Stem Cell-Derived Midbrain Dopamine Progenitor Product, MSK-DA01. Cell Stem Cell 2021 Feb 4;28(2):217-229.e7. doi: 10.1016/j.stem.2021.01.004.
Pellegrini G, Traverso CE, Franzi AT, Zingirian M, Cancedda R, De Luca M. Long-term restoration of damaged corneal surfaces with autologous cultivated corneal epithelium. Lancet. 1997;349(9057):990-993. doi:10.1016/S0140-6736(96)11188-0
Sankaran et al. Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A. Science 2008 Dec 19;322(5909):1839-42. doi: 10.1126/science.1165409.
Thomas ED, Lochte Hl Jr, Lu WC, Ferrebee JW. Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy. N Engl J Med. 1957;257(11):491-496. doi:10.1056/NEJM195709122571102
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References (Parkinson’s Disease):
Doi D, Magotani H, Kikuchi T, et al. Pre-clinical study of induced pluripotent stem cell-derived dopaminergic progenitor cells for Parkinson's disease. Nat Commun. 2020;11(1):3369. Published 2020 Jul 6. doi:10.1038/s41467-020-17165-w
Hallett PJ, Deleidi M, Astradsson A, et al. Successful function of autologous iPSC-derived dopamine neurons following transplantation in a non-human primate model of Parkinson's disease. Cell Stem Cell. 2015;16(3):269-274. doi:10.1016/j.stem.2015.01.018
Kim TW, Piao J, Koo SY, et al. Biphasic Activation of WNT Signaling Facilitates the Derivation of Midbrain Dopamine Neurons from hESCs for Translational Use. Cell Stem Cell. 2021;28(2):343-355.e5. doi:10.1016/j.stem.2021.01.005
Kikuchi T, Morizane A, Doi D, et al. Human iPS cell-derived dopaminergic neurons function in a primate Parkinson's disease model. Nature. 2017;548(7669):592-596. doi:10.1038/nature23664
López-Ornelas A, Escobedo-Avila I, Ramírez-García G, et al. Human Embryonic Stem Cell-Derived Immature Midbrain Dopaminergic Neurons Transplanted in Parkinsonian Monkeys. Cells. 2023;12(23):2738. Published 2023 Nov 30. doi:10.3390/cells12232738
Parmar M, Björklund A. From Skin to Brain: A Parkinson's Disease Patient Transplanted with His Own Cells. Cell Stem Cell. 2020;27(1):8-10. doi:10.1016/j.stem.2020.06.008
Piao J, Zabierowski S, Dubose BN, et al. Preclinical Efficacy and Safety of a Human Embryonic Stem Cell-Derived Midbrain Dopamine Progenitor Product, MSK-DA01. Cell Stem Cell. 2021;28(2):217-229.e7. doi:10.1016/j.stem.2021.01.004
Skidmore S, Barker RA. Challenges in the clinical advancement of cell therapies for Parkinson's disease. Nat Biomed Eng. 2023;7(4):370-386. doi:10.1038/s41551-022-00987-y
Takahashi J. Clinical Trial for Parkinson's Disease Gets a Green Light in the US. Cell Stem Cell. 2021;28(2):182-183. doi:10.1016/j.stem.2021.01.013
Tao Y, Vermilyea SC, Zammit M, et al. Autologous transplant therapy alleviates motor and depressive behaviors in parkinsonian monkeys. Nat Med. 2021;27(4):632-639. doi:10.1038/s41591-021-01257-1
Vermilyea SC, Emborg ME. The role of nonhuman primate models in the development of cell-based therapies for Parkinson's disease. J Neural Transm (Vienna). 2018;125(3):365-384. doi:10.1007/s00702-017-1708-9
References (AMD):
Woogeng IN, Kaczkowski B, Abugessaisa I, Hu H, Tachibana A, Sahara Y, Hon CC, Hasegawa A, Sakai N, Nishida M, Sanyal H, Sho J, Kajita K, Kasukawa T, Takasato M, Carninci P, Maeda A, Mandai M, Arner E, Takahashi M, Kime C. Inducing human retinal pigment epithelium-like cells from somatic tissue. Stem Cell Reports. 2022 Feb 8;17(2):289-306. doi: 10.1016/j.stemcr.2021.12.008. Epub 2022 Jan 13. PMID: 35030321; PMCID: PMC8828536.
McGill TJ, Bohana-Kashtan O, Stoddard JW, Andrews MD, Pandit N, Rosenberg-Belmaker LR, Wiser O, Matzrafi L, Banin E, Reubinoff B, Netzer N, Irving C. Long-Term Efficacy of GMP Grade Xeno-Free hESC-Derived RPE Cells Following Transplantation. Transl Vis Sci Technol. 2017 Jun 14;6(3):17. doi: 10.1167/tvst.6.3.17. PMID: 28626601; PMCID: PMC5472365.
References (Spinal Cord Injury):
Keirstead HS, Nistor G, Bernal G, et al. Human embryonic stem cell-derived oligodendrocyte progenitor cell transplants remyelinate and restore locomotion after spinal cord injury. J Neurosci. 2005;25(19):4694-4705. doi:10.1523/JNEUROSCI.0311-05.2005
Kobayashi Y, Okada Y, Itakura G, Iwai H, Nishimura S, Yasuda A, Nori S, Hikishima K, Konomi T, Fujiyoshi K, Tsuji O, Toyama Y, Yamanaka S, Nakamura M, Okano H. Pre-evaluated safe human iPSC-derived neural stem cells promote functional recovery after spinal cord injury in common marmoset without tumorigenicity. PLoS One. 2012;7(12):e52787. doi: 10.1371/journal.pone.0052787
Nori S, Okada Y, Yasuda A, et al. Grafted human-induced pluripotent stem-cell-derived neurospheres promote motor functional recovery after spinal cord injury in mice. Proc Natl Acad Sci U S A. 2011;108(40):16825-16830. doi:10.1073/pnas.1108077108
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References:
Abou-El-Enein M, Römhild A, Kaiser D, et al. Good Manufacturing Practices (GMP) manufacturing of advanced therapy medicinal products: a novel tailored model for optimizing performance and estimating costs. Cytotherapy. 2013;15(3):362-383. doi:10.1016/j.jcyt.2012.09.006
Abou-El-Enein M, Elsallab M, Feldman SA, et al. Scalable Manufacturing of CAR T cells for Cancer Immunotherapy. Blood Cancer Discov. 2021;2(5):408-422. doi:10.1158/2643-3230.BCD-21-0084
Dashnau JL, Xue Q, Nelson M, Law E, Cao L, Hei D. A risk-based approach for cell line development, manufacturing and characterization of genetically engineered, induced pluripotent stem cell-derived allogeneic cell therapies. Cytotherapy. 2023;25(1):1-13. doi:10.1016/j.jcyt.2022.08.001
Madrid M, Sumen C, Aivio S, Saklayen N. Autologous Induced Pluripotent Stem Cell-Based Cell Therapies: Promise, Progress, and Challenges. Curr Protoc. 2021;1(3):e88. doi:10.1002/cpz1.88
National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Sciences Policy; Forum on Regenerative Medicine. Navigating the Manufacturing Process and Ensuring the Quality of Regenerative Medicine Therapies: Proceedings of a Workshop. Washington (DC): National Academies Press (US); 2017 Oct 26. 3, Identifying and Measuring Critical Quality Attributes. Available from: https://www.ncbi.nlm.nih.gov/books/NBK475684/
Salmikangas P, Carlsson B, Klumb C, Reimer T, Thirstrup S. Potency testing of cell and gene therapy products. Front Med (Lausanne). 2023;10:1190016. Published 2023 May 5. doi:10.3389/fmed.2023.1190016
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References:
Stem Cell Translation
Pellegrini G, Lambiase A, Macaluso C, et al. From discovery to approval of an advanced therapy medicinal product-containing stem cells, in the EU. Regen Med. 2016;11(4):407-420. doi:10.2217/rme-2015-0051
Sugai K, Sumida M, Shofuda T, et al. First-in-human clinical trial of transplantation of iPSC-derived NS/PCs in subacute complete spinal cord injury: Study protocol. Regen Ther. 2021;18:321-333. Published 2021 Sep 7. doi:10.1016/j.reth.2021.08.005
Compassionate Use
Hirsch T, Rothoeft T, Teig N, et al. Regeneration of the entire human epidermis using transgenic stem cells. Nature. 2017;551(7680):327-332. doi:10.1038/nature24487
Reverse Translation:
Rama P, Matuska S, Paganoni G, Spinelli A, De Luca M, Pellegrini G. Limbal stem-cell therapy and long-term corneal regeneration. N Engl J Med. 2010;363(2):147-155. doi:10.1056/NEJMoa0905955
Pellegrini G, Rama P, Matuska S, et al. Biological parameters determining the clinical outcome of autologous cultures of limbal stem cells. Regen Med. 2013;8(5):553-567. doi:10.2217/rme.13.43
Real World Evidence:
Wang SV, Schneeweiss S; RCT-DUPLICATE Initiative, et al. Emulation of Randomized Clinical Trials With Nonrandomized Database Analyses: Results of 32 Clinical Trials [published correction appears in JAMA. 2024 Apr 9;331(14):1236. doi: 10.1001/jama.2024.5022.]. JAMA. 2023;329(16):1376-1385. doi:10.1001/jama.2023.4221
Preclinical Studies
Barker RA; TRANSEURO consortium. Designing stem-cell-based dopamine cell replacement trials for Parkinson's disease. Nat Med. 2019;25(7):1045-1053. doi:10.1038/s41591-019-0507-2
Kirkeby A, Nelander J, Hoban DB, et al. Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD. Cell Stem Cell. 2023;30(10):1299-1314.e9. doi:10.1016/j.stem.2023.08.014
Kirkeby A, Main H, Carpenter M. Pluripotent stem-cell-derived therapies in clinical trial: A 2025 update. Cell Stem Cell. 2025;32(2):329-331. doi:10.1016/j.stem.2025.01.003
Additional References:
Barker RA, Carpenter M, Jamieson CHM, et al. Lessons learnt, and still to learn, in first in human stem cell trials. Stem Cell Reports. 2023;18(8):1599-1609. doi:10.1016/j.stemcr.2022.11.019
da Cruz L, Fynes K, Georgiadis O, et al. Phase 1 clinical study of an embryonic stem cell-derived retinal pigment epithelium patch in age-related macular degeneration. Nat Biotechnol. 2018;36(4):328-337. doi:10.1038/nbt.4114
Lovell-Badge R, Anthony E, Barker RA, et al. ISSCR Guidelines for Stem Cell Research and Clinical Translation: The 2021 update. Stem Cell Reports. 2021;16(6):1398-1408. doi:10.1016/j.stemcr.2021.05.012
Pellegrini G, Ardigò D, Milazzo G, et al. Navigating Market Authorization: The Path Holoclar Took to Become the First Stem Cell Product Approved in the European Union. Stem Cells Transl Med. 2018;7(1):146-154. doi:10.1002/sctm.17-0003
Sugarman J, Barker RA, Kerridge I, et al. Tackling Ethical Challenges of Premature Delivery of Stem Cell-Based Therapies: ISSCR 2018 Annual Meeting Focus Session Report. Stem Cell Reports. 2018;11(5):1021-1025. doi:10.1016/j.stemcr.2018.10.020
Sugarman J, Barker RA, Charo RA. A Professional Standard for Informed Consent for Stem Cell Therapies. JAMA. 2019;322(17):1651-1652. doi:10.1001/jama.2019.11290
WMA Declaration of Helsinki – Ethical Principles for Medical Research Involving Human Participants
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References:
Regulatory:
Ikonomou L, Munsie M, Power C, Sipp D, Turner L, Rasko JEJ. Effective regulatory responses to predatory stem cell markets in Australia and Canada. Cell Stem Cell. 2024;31(10):1393-1397. doi:10.1016/j.stem.2024.09.007
Lindvall O. and Hyun I., Medical innovation versus stem cell tourism,” Science. 2009;324(5935):1664-1665. doi: 10.1126/science.1171749
Matthews KRW, Lowe SJ, Master Z. US state laws on medical freedom and investigational stem cell procedures: a call to focus on state-based legislation. Cytotherapy. 2024;26(4):404-409. doi:10.1016/j.jcyt.2024.01.002
Master Z, Matthews KRW, Abou-El-Enein M. Unproven stem cell interventions: A global public health problem requiring global deliberation. Stem Cell Reports. 2021;16(6):1435-1445. doi:10.1016/j.stemcr.2021.05.004
Munsie M, Hyun I. A question of ethics: selling autologous stem cell therapies flaunts professional standards. Stem Cell Res. 2014;13(3 Pt B):647-653. doi:10.1016/j.scr.2014.04.014
Pew Charitable Trusts. 2019. FDA’s Framework for Regulating Regenerative Medicine Will Improve Oversight. https://www.pewtrusts.org/en/research-and-analysis/reports/2019/10/17/fdas-framework-for-regulating-regenerative-medicine-will-improve-oversight.
Richardson E, Akkas F, Master Z. Evaluating the FDA regenerative medicine framework: opportunities for stakeholders. Regen Med. 2020;15(7):1825-1832. doi: 10.2217/rme-2020-0073.
Rudge C, Ghinea N, Munsie M, Stewart C. Regulating autologous stem cell interventions in Australia: updated review of the direct-to-consumer advertising restrictions. Aust Health Rev. 2021;45(4):507-515. doi:10.1071/AH20217
Zarzeczny A, Caulfield T, Ogbogu U, et al. Professional regulation: a potentially valuable tool in responding to "stem cell tourism". Stem Cell Reports. 2014;3(3):379-384. doi:10.1016/j.stemcr.2014.06.016
Patient Perceptions:
Arthurs JR, Nordan LM, Hultgren BH, et al. Patients seeking stem cell therapies-a prospective qualitative analysis from a Regenerative Medicine Consult Service [published correction appears in NPJ Regen Med. 2023 Feb 2;8(1):5. doi: 10.1038/s41536-023-00278-3.]. NPJ Regen Med. 2022;7(1):20. Published 2022 Mar 25. doi:10.1038/s41536-022-00215-w
Kawam, O., Zhu, X., Watson, S., Martin-Lillie, C., Arthurs, J., Eton, D.T., Rutten, L.J.F., Shapiro, S., Tilburt, J.C. and Master, Z. 2025. Factors that influence unproven stem cell intervention seeking behavior: a qualitative analysis of U.S. patients considering or having taken unproven stem cell interventions. Social Science & Medicine. In press.v
Smith C, Martin-Lillie C, Higano JD, et al. Challenging misinformation and engaging patients: characterizing a regenerative medicine consult service. Regen Med. 2020;15(3):1427-1440. doi:10.2217/rme-2020-0018
Tanner C, Petersen A, Munsie M. 'No one here's helping me, what do you do?': addressing patient need for support and advice about stem cell treatments. Regen Med. 2017;12(7):791-801. doi:10.2217/rme-2017-0056
Waldby C, Hendl T, Kerridge I, et al. The direct-to-consumer market for stem cell-based interventions in Australia: exploring the experiences of patients. Regen Med. 2020;15(1):1238-1249. doi:10.2217/rme-2019-0089
Zarzeczny A, Tanner C, Barfoot J, Blackburn C, Couturier A, Munsie M. Contact us for more information: an analysis of public enquiries about stem cells. Regen Med. 2019;14(12):1137-1150. doi:10.2217/rme-2019-0092
Physician Perceptions and Duties:
Arthurs JR, Martin Lillie CM, Master Z, Shapiro SA. The Direct to Consumer Stem Cell Market and the Role of Primary Care Providers in Correcting Misinformation. J Prim Care Community Health. 2022;13:21501319221121460. doi:10.1177/21501319221121460
Bowman M, Racke M, Kissel J, Imitola J. Responsibilities of Health Care Professionals in Counseling and Educating Patients With Incurable Neurological Diseases Regarding "Stem Cell Tourism": Caveat Emptor. JAMA Neurol. 2015;72(11):1342-1345. doi:10.1001/jamaneurol.2015.1891
Fu W, Smith C, Turner L, Fojtik J, Pacyna JE, Master Z. Characteristics and Scope of Training of Clinicians Participating in the US Direct-to-Consumer Marketplace for Unproven Stem Cell Interventions. JAMA. 2019;321(24):2463-2464. doi:10.1001/jama.2019.5837
Julian K, Yuhasz N, Rai W, Salerno JA, Imitola J. Complications from "Stem Cell Tourism" in Neurology. Ann Neurol. 2020;88(4):661-668. doi:10.1002/ana.25842
Matthews KRW, Kawam O, Sprung K, Perin EC, Master Z. Role of Physicians in Guiding Patients Away From Unproven Stem Cell Interventions. Tex Heart Inst J. 2022;49(2):e217768. doi:10.14503/THIJ-21-7768
Rees L, Friis T, Woodward-Kron R, Munsie M. What is known about healthcare professional-patient communication when discussing stem cell therapies? A scoping review. Patient Educ Couns. 2025;130:108430. doi:10.1016/j.pec.2024.108430
Smith C, Crowley A, Munsie M, et al. Academic physician specialists' views toward the unproven stem cell intervention industry: areas of common ground and divergence. Cytotherapy. 2021;23(4):348-356. doi:10.1016/j.jcyt.2020.12.011
Marketing:
Chavez J, Shah NA, Ruoss S, Cuomo RE, Ward SR, Mackey TK. Online marketing practices of regenerative medicine clinics in US-Mexico border region: a web surveillance study. Stem Cell Res Ther. 2021;12(1):189. Published 2021 Mar 18. doi:10.1186/s13287-021-02254-4
Cook M, Richey A, Brafman DA, Frow EK. Weighing up the evidence used by direct-to-consumer stem cell businesses. Stem Cell Reports. 2021;16(12):2852-2860. doi:10.1016/j.stemcr.2021.10.007
Hawke B, Przybylo AR, Paciulli D, Caulfield T, Zarzeczny A, Master Z. How to Peddle Hope: An Analysis of YouTube Patient Testimonials of Unproven Stem Cell Treatments. Stem Cell Reports. 2019;12(6):1186-1189. doi:10.1016/j.stemcr.2019.05.009
Sipp D, Caulfield T, Kaye J, et al. Marketing of unproven stem cell-based interventions: A call to action. Sci Transl Med. 2017;9(397):eaag0426. doi:10.1126/scitranslmed.aag0426
Turner L. The American stem cell sell in 2021: U.S. businesses selling unlicensed and unproven stem cell interventions. Cell Stem Cell. 2021;28(11):1891-1895. doi:10.1016/j.stem.2021.10.008
Turner L, Martinez JR Jr, Najjar S, Rajapaksha Arachchilage T, Wang JC. Businesses marketing purported stem cell treatments and exosome therapies for COVID-19: An analysis of direct-to-consumer online advertising claims. Stem Cell Reports. 2023;18(11):2010-2015. doi:10.1016/j.stemcr.2023.09.015
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References:
Bowman M, Racke M, Kissel J, Imitola J. Responsibilities of Health Care Professionals in Counseling and Educating Patients With Incurable Neurological Diseases Regarding "Stem Cell Tourism": Caveat Emptor. JAMA Neurol. 2015;72(11):1342-1345. doi:10.1001/jamaneurol.2015.1891
Feudtner C. The breadth of hopes. N Engl J Med. 2009;361(24):2306-2307. doi:10.1056/NEJMp0906516
Hyun I. Therapeutic hope, spiritual distress, and the problem of stem cell tourism. Cell Stem Cell. 2013;12(5):505-507. doi:10.1016/j.stem.2013.04.010
Julian K, Yuhasz N, Hollingsworth E, Imitola J. The "Growing" Reality of the Neurological Complications of Global "Stem Cell Tourism". Semin Neurol. 2018;38(2):176-181. doi:10.1055/s-0038-1649338
Julian K, Yuhasz N, Rai W, Salerno JA, Imitola J. Complications from "Stem Cell Tourism" in Neurology. Ann Neurol. 2020;88(4):661-668. doi:10.1002/ana.25842
Kawam O, Zhu X, Eton DT, et al. Designing persuasive health education for patients seeking unproven stem cell interventions. Stem Cell Reports. 2023;18(8):1549-1554. doi:10.1016/j.stemcr.2023.06.004
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