References
- Henig I, Zuckerman T. Hematopoietic stem cell transplantation—50 years of evolution and future perspectives. Rambam Maimonides Med J. 2014;5(4):e0028.
- Copelan EA. Hematopoietic stem-cell transplantation. N Engl J Med. 2006;354(17):1813–1826.
- Niederwieser D, Baldomero H, Atsuta Y, et al. One and half million Hematopoietic Stem Cell Transplants (HSCT). Dissemination, trends and potential to improve activity by telemedicine from the Worldwide Network for Blood and Marrow Transplantation (WBMT). Blood. 2019;134(Supplement_1):2035.
- Dykes JH, Toporski J, Juliusson G, et al. Rapid and effective CD3 T-cell depletion with a magnetic cell sorting program to produce peripheral blood progenitor cell products for haploidentical transplantation in children and adults. Transfusion (Paris). 2007;47(11):2134–2142.
- Berenson RJ, Andrews RG, Bensinger WI, et al. Antigen CD34+ marrow cells engraft lethally irradiated baboons. J Clin Invest. 1988;81(3):951–955.
- Berenson R, Bensinger W, Hill R, et al. Engraftment after infusion of CD34+ marrow cells in patients with breast cancer or neuroblastoma. Blood. 1991;77(8):1717–1722.
- Lee-Six H, Kent DG. Tracking hematopoietic stem cells and their progeny using whole-genome sequencing. Exp Hematol. 2020;83:12–24.
- Bordignon C, Notarangelo LD, Nobili N, et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA − immunodeficient patients. Science. 1995;270(5235):470–475.
- Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 2002;296(5577):2410–2413.
- Cavazzana-Calvo M, Hacein-Bey S, Basile GDS, et al. Gene therapy of human Severe Combined Immunodeficiency (SCID)-X1 disease. Science. 2000;288(5466):669–672.
- Gaspar HB, Parsley KL, Howe S, et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet. 2004;364(9452):2181–2187.
- Alliance for Regenerative Medicine. Available Products [Internet]. [cited 2022 Jun 19]. Available from: https://alliancerm.org/available-products
- Li M, Kassim S. Decentralized manufacturing: from stem cell transplants to the next generation of cellular immunotherapies. Cell Gene Ther Insights. 2020;6(6):697–714.
- Allocord. Package Insert [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/86181/download
- Clevecord. Package Insert [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/99648/download.
- Ducord. Package Insert [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/84567/download.
- Hemacord. Package Insert [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/82016/download.
- University of Colorado Cord BLood Bank. HPC Cord Blood, Package Insert [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/83601/download.
- MD Anderson Cord Blood Bank. HPC Cord Blood, Package Insert [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/114119/download.
- Bloodworks. HPC Cord Blood, Package Insert [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/95521/download.
- Bluebird Bio. Skysona, product information [Internet]. [cited 2022 Jun 19]. Available from: https://www.ema.europa.eu/en/documents/product-information/skysona-epar-product-information_en.pdf.
- Orchard Therapeutics. Strimvelis, Product Information [Internet]. [cited 2022 Jun 19]. Available from: https://www.ema.europa.eu/en/documents/product-information/strimvelis-epar-product-information_en.pdf
- Bluebird BIo. Zynteglo, Product Information [Internet]. [cited 2022 Jun 19]. Available from: https://www.ema.europa.eu/en/documents/product-information/zynteglo-epar-product-information_en.pdf
- 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.
- Xu L, Wang J, Liu Y, et al. CRISPR-edited stem cells in a patient with HIV and acute lymphocytic leukemia. N Engl J Med. 2019;381(13):1240–1247.
- Bluebird Bio. elivaldogene autotemcel Advisory Committee Briefing Document [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/159012/download.
- Vor Biopharma. Vor announces FDA clearance of IND Application for VOR33 [Internet]. [cited 2022 Jun 19]. Available from: https://www.vorbio.com/vor-announces-fda-clearance-of-ind-application-for-vor33
- Gamida Cell. Pipeline [Internet]. [cited 2022 Jun 19]. Available from: https://www.gamida-cell.com/our-rd/
- Faulkner S. Gamida cell touts first patient treated in Phase III NiCord trial [Internet]. Drug Deliv. Bus. [cited 2022 Jun 19]. Available from: https://www.drugdeliverybusiness.com/gamida-cell-touts-first-patient-treated-phase-iii-nicord-trial/
- ClinicalTrials.gov. Gene correction in autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease (CEDAR) [Internet]. [cited 2022 Jun 19]. Available from: https://clinicaltrials.gov/ct2/show/NCT04819841?term=graphite&draw=2
- Graphite BIo. Our science: overview [Internet]. [cited 2022 Jun 19]. Available from: https://graphitebio.com/our-science
- Orca Bio. Pipeline [Internet]. [cited 2022 Jun 19]. Available from: https://orcabio.com/patients
- Plieth J. Glaxo’s gene therapy exit prompts truce with Orchard [Internet]. Evaluate. [cited 2022 Jun 19]. Available from: https://www.evaluate.com/jp/node/13005
- Orchard Therapeutics Unveils Details on New HSC Gene Therapy Research Programs as Part of R&D Investor Event Tomorrow at 9:00 a.m. ET [Internet]. Globe Newswire. [cited 2022 Jun 19]. Available from: https://www.globenewswire.com/en/news-release/2020/11/12/2126007/0/en/Orchard-Therapeutics-Unveils-Details-on-New-HSC-Gene-Therapy-Research-Programs-as-Part-of-R-D-Investor-Event-Tomorrow-at-9-00-a-m-ET.html
- Vor Biopharma. Pipeline [Internet]. [cited 2022 Jun 19]. Available from: https://www.ema.europa.eu/en/documents/product-information/zynteglo-epar-product-information_en.pdf.
- Soni S, Kohn DB. Chemistry, manufacturing and controls for gene modified hematopoietic stem cells. Cytotherapy. 2019;21(3):358–366.
- Champlin R. Selection of autologous or allogeneic transplantation. In: Kufe DW, Pollock RE, Weichselbaum RR, et al., editors. Holland-Frei Cancer Medicine. 6th ed. Hamilton (ON): BC Decker; 2003.
- Thomas ED, Storb R, Clift RA, et al. Bone-marrow transplantation: (First of two parts). N Engl J Med. 1975;292(16):832–843.
- Thomas ED, Storb R, Clift RA, et al. Bone-marrow transplantation: (Second of two parts). N Engl J Med. 1975;292(17):895–902.
- Tuthill M, and Hatzimichael. Hematopoietic stem cell transplantation. Stem Cells Cloning Adv Appl. 2010;105:105–117.
- Körbling M, Freireich EJ. Twenty-five years of peripheral blood stem cell transplantation. Blood. 2011;117(24):6411–6416.
- Ballen KK, Gluckman E, Broxmeyer HE. Umbilical cord blood transplantation: the first 25 years and beyond. Blood. 2013;122(4):491–498.
- Pecora AL. Impact of stem cell dose on hematopoietic recovery in autologous blood stem cell recipients. Bone Marrow Transplant. 1999;23(S2):S7–S12.
- Maffini E, Labopin M, Blaise D, et al. CD34 + cell dose effects on clinical outcomes after T‐cell replete haploidentical allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia using peripheral blood stem cells. A study from the acute leukemia working Party of the European Society for blood and marrow transplantation (EBMT). Am J Hematol. 2020;95(8):892–899.
- Martin PS, Li S, Nikiforow S, et al. Infused total nucleated cell dose is a better predictor of transplant outcomes than CD34 + cell number in reduced-intensity mobilized peripheral blood allogeneic hematopoietic cell transplantation. Haematologica. 2016;101(4):499–505.
- Chen BJ, Cui X, Sempowski GD, et al. Hematopoietic stem cell dose correlates with the speed of immune reconstitution after stem cell transplantation. Blood. 2004;103(11):4344–4352.
- Remberger M, Törlén J, Ringdén O, et al. Effect of total nucleated and CD34+ cell dose on outcome after allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2015;21(5):889–893.
- Heimfeld S. Bone marrow transplantation: how important is CD34 cell dose in HLA-identical stem cell transplantation? Leukemia. 2003;17(5):856–858.
- Mehta J, Mehta J, Frankfurt O, et al. Optimizing the CD34 + cell dose for reduced-intensity allogeneic hematopoietic stem cell transplantation. Leuk Lymphoma. 2009;50(9):1434–1441.
- Hübel K. Mobilization and Collection of HSC. Carreras E, Dufour C, Mohty M, et al. editors. EBMT Handb [Internet]. Cham: Springer International Publishing; 2019; p. 117–122. [cited 2022 Jun 19]. Available from: http://link.springer.com/10.1007/978-3-030-02278-5_15
- Bilgin YM. Use of plerixafor for stem cell mobilization in the setting of autologous and allogeneic stem cell transplantations: an update. J Blood Med. 2021;12:403–412.
- Miller HK, Braun TM, Stillwell T, et al. Infectious risk after allogeneic hematopoietic cell transplantation complicated by acute graft-versus-host disease. Biol Blood Marrow Transplant. 2017;23(3):522–528.
- Khaddour K, Hana C, and Mewawalla P. Hematopoietic stem cell transplantation. Treasure Island, Florida, USA: StatPearls HQ; 2022.
- CellGenix. SCGM data sheet [Internet]. [cited 2022 Jun 19]. Available from: https://cellgenix.com/wp-content/uploads/2021/11/Data-Sheet_GMP-SCGM_ME-DS-2002e.pdf.
- Lonza. TheraPEAK X-VIVO-10 Serum-free Hematopoietic Cell Medium [Internet]. [cited 2022 Jun 19]. Available from: https://bioscience.lonza.com/lonza_bs/CH/en/Culture-Media-and-Reagents/p/000000000000216937/TheraPEAK-X-VIVO-10-Serum-free-Hematopoietic-Cell-Medium#
- Osawa M, Hanada K, Hamada H, et al. Long-Term Lymphohematopoietic Reconstitution by a Single CD34-Low/Negative Hematopoietic Stem Cell. Science. 1996;273(5272):242–245.
- Goodell MA. Introduction: focus on hematology. CD34(+) or CD34(-): does it really matter? Blood. 1999;94(8):2545–2547.
- Yadav P, Vats R, Bano A, et al. Hematopoietic stem cells culture, expansion and differentiation: an insight into variable and available media. Int J Stem Cells. 2020;13(3):326–334.
- Dooley DC, Oppenlander BK, Xiao M. Analysis of Primitive CD34 − and CD34 + Hematopoietic Cells from Adults: gain and Loss of CD34 Antigen by Undifferentiated Cells Are Closely Linked to Proliferative Status in Culture. Stem cells. 2004;22(4):556–569.
- Caux C, Favre C, Saeland S, et al. Sequential loss of CD34 and class II MHC antigens on purified cord blood hematopoietic progenitors cultured with IL-3: characterization of CD34-, HLA-DR+ cells. Blood. 1989;74(4):1287–1294.
- Mesquitta W-T, Wandsnider M, Kang H, et al. UM171 expands distinct types of myeloid and NK progenitors from human pluripotent stem cells. Sci Rep. 2019;9(1):6622.
- Papa L, Djedaini M, Hoffman R. Ex vivo HSC expansion challenges the paradigm of unidirectional human hematopoiesis. Ann N Y Acad Sci. 2020;1466(1):39–50.
- O’Leary HA. “Breaking down” the mechanisms of expansion. Blood. 2020;136(19):2095–2096.
- Mehta RS, Rezvani K, Olson A, et al. Novel techniques for ex vivo expansion of cord blood: clinical trials. Front Med [Internet]. 2015;2. DOI:10.3389/fmed.2015.00089.
- Bari S, Zhong Q, Fan X, et al. Ex vivo expansion of CD34+CD90+CD49f+ hematopoietic stem and progenitor cells from non-enriched umbilical cord blood with azole compounds. Stem Cells Transl Med. 2018;7:376–393.
- Meneghel J, Kilbride P, Morris GJ. Cryopreservation as a key element in the successful delivery of cell-based therapies—A review. Front Med. 2020;7:592242.
- Alcorn MJ, Holyoake TL, Richmond LJ, et al. CD34+ cells can be selected efficiently from cryopreserved peripheral blood progenitor cells and can retain their proliferative potential. J Hematother. 1997;6(5):501–510.
- Galmes A, Gutierrez A, Sampol A, et al. Long-term hematologic reconstitution and clinical evaluation of autologous peripheral blood stem cell transplantation after cryopreservation of cells with 5% and 10% dimethylsulfoxide at 80 C in a mechanical freezer. Haematologica. 2007;92(7):986–989.
- Rowley SD, Feng Z, Chen L, et al. A randomized phase III clinical trial of autologous blood stem cell transplantation comparing cryopreservation using dimethylsulfoxide vs dimethylsulfoxide with hydroxyethylstarch. Bone Marrow Transplant. 2003;31(11):1043–1051.
- Bulcha JT, Wang Y, Ma H, et al. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021;6(1):53.
- Ghosh S, Brown AM, Jenkins C, et al. Viral vector systems for gene therapy: a comprehensive literature review of progress and biosafety challenges. Appl Biosaf. 2020;25(1):7–18.
- Li H, Yang Y, Hong W, et al. Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects. Signal Transduct Target Ther. 2020;5(1):1.
- Khalil AM. The genome editing revolution: review. J Genet Eng Biotechnol. 2020;18(1):68.
- Anzalone AV, Koblan LW, Liu DR. Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors. Nat Biotechnol. 2020;38(7):824–844.
- Carmen J. Merits of non-viral cellular engineering versus viral cellular engineering. Cell Gene Ther Insights. 2018;4(2):113–117.
- Croop J, Cooper R, Seshadri R, et al. Large-scale mobilization and isolation of CD34+ cells from normal donors. Bone Marrow Transplant. 2000;26(12):1271–1279.
- Fu P, Bagai RK, Meyerson H, et al. Pre-mobilization therapy blood CD34+ cell count predicts the likelihood of successful hematopoietic stem cell mobilization. Bone Marrow Transplant. 2006;38(3):189–196.
- Sutherland DR, Keating A.The CD34 antigen: structure, biology, and potential clinical applications.J Hematother.1992;1:115–129.
- Sutherland DR, Stewart AK, Keating A. CD34 antigen: molecular features and potential clinical applications. Stem Cells. 1993;11(S3):50–57.
- Holyoake TL, Alcorn MJ, Franklin IM. The CD34 antigen: potential clinical advantages of CD34 selection. Clin Oncol. 1996;8(4):214–221.
- Creative Bioarray. SuperBeads® Human Whole Blood CD34+ Cell Isolation Kit [Internet]. [cited 2022 Jun 19]. Available from: https://www.creative-bioarray.com/superbeads-human-whole-blood-cd34-cell-isolation-kit-cat-csk-ci008-item-1065.htm
- Biotec M. Automated cell separation systems [Internet]. [cited 2022 Jun 19]. Available from: https://www.miltenyibiotec.com/US-en/products/macs-cell-separation/instruments.html?countryRedirected=1
- Zborowski M, and Chalmers J. Magnetic cell separation. Amsterdam, Netherlands: Elsevier Science; 2007.
- Stemcell Technologies. EasySepTM Cell Separation Magnets [Internet]. [cited 2022 Jun 19]. Available from: https://www.stemcell.com/easysep-cell-separation-magnets.html
- Stemcell Technologies. Instruments and Software [Internet]. [cited 2022 Jun 19]. Available from: https://www.stemcell.com/products/product-types/instruments.html
- Neurauter AA, Bonyhadi M, Lien E, et al. Cell isolation and expansion using Dynabeads ®.In: Kumar A, Galaev IY, Mattiasson B, editors. Cell Sep [Internet]. Berlin Heidelberg: Springer Berlin Heidelberg; 2008. p. 41–73. [cited 2022 Jun 19]. Available from: http://link.springer.com/10.1007/10_2007_072
- Thermo Fisher Scientific. Magnets for Molecular and Cell Separation Applications [Internet]. [cited 2022 Jun 19]. Available from: https://www.thermofisher.com/us/en/home/brands/product-brand/dynal/magnets.html
- Millington M, Arndt A, Boyd M, et al. Towards a Clinically Relevant Lentiviral Transduction Protocol for Primary Human CD34+ Hematopoietic Stem/Progenitor Cells. Giannobile W, editor. PLoS ONE. 2009;4:e6461.
- Schott JW, León-Rico D, Ferreira CB, et al. Enhancing lentiviral and alpharetroviral transduction of human hematopoietic stem cells for clinical application. Mol Ther - Methods Clin Dev. 2019;14:134–147.
- Shapiro J, Iancu O, Jacobi AM, et al. Increasing CRISPR efficiency and measuring its specificity in HSPCs using a clinically relevant system. Mol Ther - Methods Clin Dev. 2020;17:1097–1107.
- Prakash V, Moore M, Yáñez-Muñoz RJ. Current progress in therapeutic gene editing for monogenic diseases. Mol Ther. 2016;24(3):465–474.
- Lonza. Transfection [Internet]. [cited 2022 Jun 19]. Available from: https://bioscience.lonza.com/lonza_bs/US/en/Catalogue/Products/Transfection/c/6
- Lonza. GMP solutions for cell and gene therapy [Internet]. [cited 2022 Jun 19]. Available from: https://bioscience.lonza.com/lonza_bs/US/en/gmp-solutions-for-cell-and-gene-therapy
- Zonari E, Desantis G, Petrillo C, et al. Efficient ex vivo engineering and expansion of highly purified human hematopoietic stem and progenitor cell populations for gene therapy. Stem Cell Rep. 2017;8(4):977–990.
- MaxCyte. Processing assemblies accessories [Internet]. [cited 2022 Jun 19]. Available from: https://maxcyte.com/pa-accessories/.
- Thermo Fisher Scientific. Electroporation Transformation Kits [Internet]. [cited 2022 Jun 19]. Available from: https://www.thermofisher.com/search/browse/category/us/en/90220064/electroporation+transformation+kits.
- Thermo Fisher Scientific. CTS Xenon Electroporation Instrument [Internet]. [cited 2022 Jun 19]. Available from: https://assets.thermofisher.com/TFS-Assets/BID/Flyers/cts-xenon-electroporation-system-flyer.pdf.
- Thermo Fisher Scientific. Neon(TM) Transfection System Starter Pack [Internet]. [cited 2022 Jun 19]. Available from: https://www.thermofisher.com/order/catalog/product/MPK5000S.
- Biolife Solutions. Programmable Controlled Rate Freezers [Internet]. [cited 2022 Jun 19]. Available from: https://www.biolifesolutions.com/cryogenic-storage/controlled-rate-freezers/.
- Planer. Cryogenic Freezers [Internet]. [cited 2022 Jun 19]. Available from: https://planer.com/products/cryo-freezers.html.
- Morgenstern DA, Ahsan G, Brocklesby M, et al. Post-thaw viability of cryopreserved peripheral blood stem cells (PBSC) does not guarantee functional activity: important implications for quality assurance of stem cell transplant programmes. Br J Haematol. 2016;174(6):942–951.
- Kilbride P, Meneghel J, Lamb S, et al. Recovery and post-thaw assessment of human umbilical cord blood cryopreserved as quality control segments and bulk samples. Biol Blood Marrow Transplant. 2019;25(12):2447–2453.
- Cytiva. VIA freeze controlled-rate freezers [Internet]. [cited 2022 Jun 19]. Available from: https://www.cytivalifesciences.com/en/us/shop/cell-therapy/systems/via-freeze-controlled-rate-freezers-p-09721.
- McNIECE IK, Stoney GB, Kern BP, et al. CD34+ cell selection from frozen cord blood products using the isolex 300i and CliniMACS CD34 selection devices. J Hematother. 1998;7(5):457–461.
- Mantha S, Savage DG, Nichols GL, et al. CD34+ selection before autologous peripheral blood stem cell transplantation: comparison of the Nexell isolex 300i® and miltenyi biotec CliniMACS® systems. Blood. 2005;106(11):1070.
- Bornhäuser M, Platzbecker U, Theuser C, et al. CD34 + -enriched peripheral blood progenitor cells from unrelated donors for allografting of adult patients: high risk of graft failure, infection and relapse despite donor lymphocyte add-back: allogeneic Transplantation of CD34 + PBPC from Unrelated Donors. Br J Haematol. 2002;118(4):1095–1103.
- Elmaagacli AH, Peceny R, Steckel N, et al. Outcome of transplantation of highly purified peripheral blood CD34+ cells with T-cell add-back compared with unmanipulated bone marrow or peripheral blood stem cells from HLA-identical sibling donors in patients with first chronic phase chronic myeloid leukemia. Blood. 2003;101(2):446–453.
- Stainer CJ, Miflin G, Anderson S, et al. A comparison of two different systems for CD34+ selection of autologous or allogeneic PBSC collections. J Hematother. 1998;7. 375–383.
- O’Donnell PV, Myers B, Edwards J, et al. CD34 selection using three immunoselection devices: comparison of T-cell depleted allografts. Cytotherapy. 2001;3(6):483–488.
- Bjorkstrand B, Sundman-Engberg B, Christensson B, et al. A controlled comparison of two different clinical grade devices for CD34+ cell selection of autologous blood stem cell grafts. J Hematother. 1999;8(1):75–80.
- Antonenas V. Results of using automated CliniMACS prodigy for CD34 selection from mobilized peripheral blood stem cell products. Cytotherapy. 2018;20(5):e4.
- Hümmer C, Poppe C, Bunos M, et al. Automation of cellular therapy product manufacturing: results of a split validation comparing CD34 selection of peripheral blood stem cell apheresis product with a semi-manual vs. an automatic procedure. J Transl Med. 2016;14(1):76.
- Clarke E, Potter M, Oakhill A, et al. A laboratory comparison of T cell depletion by CD34+ cell immunoaffinity selection and in vitro Campath-1M treatment: clinical implications for bone marrow transplantation and donor leukocyte therapy. Bone Marrow Transplant. 1997;20(7):599–605.
- Spohn G, Wiercinska E, Karpova D, et al. Automated CD34+ cell isolation of peripheral blood stem cell apheresis product. Cytotherapy. 2015;17(10):1465–1471.
- Stroncek DF, Tran M, Frodigh SE, et al. Preliminary evaluation of a highly automated instrument for the selection of CD34+ cells from mobilized peripheral blood stem cell concentrates: SELECTION OF CD34+ CELLS. Transfusion (Paris). 2016;56(2):511–517.
- Li H, Luo Q, Shan W, et al. Biomechanical cues as master regulators of hematopoietic stem cell fate. Cell Mol Life Sci. 2021;78(16):5881–5902.
- Jing Q, Cai H, Du Z, et al. Effects of agitation speed on the ex vivo expansion of cord blood hematopoietic stem/progenitor cells in stirred suspension culture. Artif Cells Nanomed Biotechnol. 2013;41(2):98–102.
- Cossarizza A, Chang H, Radbruch A, et al. Guidelines for the use of flow cytometry and cell sorting in immunological studies (second edition). Eur J Immunol. 2019;49(10):1457–1973.
- Radtke S, Pande D, Cui M, et al. Purification of human CD34+CD90+ HSCs reduces target cell population and improves lentiviral transduction for gene therapy. Mol Ther - Methods Clin Dev. 2020;18:679–691.
- STgenetics. Cytonome cell sorter aids in Parkinson’s treatment clinical trial [Internet]. Globe Newswire. [cited 2022 Jun 19]. Available from: https://www.globenewswire.com/en/news-release/2021/04/19/2212452/0/en/Cytonome-cell-sorter-aids-in-Parkinson-s-treatment-clinical-trial.html.
- Bono N, Ponti F, Mantovani D, et al. Non-Viral in vitro gene delivery: it is now time to set the bar! Pharmaceutics. 2020;12(2):183.
- Yahya EB, Alqadhi AM. Recent trends in cancer therapy: a review on the current state of gene delivery. Life Sci. 2021;269:119087.
- Walasek MA, van Os R, de Haan G. Hematopoietic stem cell expansion: challenges and opportunities: HSC expansion: challenges and opportunities. Ann N Y Acad Sci. 2012;1266(1):138–150.
- Morgan RA, Gray D, Lomova A, et al. Hematopoietic stem cell gene therapy: progress and lessons learned. Cell Stem Cell. 2017;21(5):574–590.
- Purdy MH, Hogan CJ, Hami L, et al. Large volume ex vivo expansion of CD34-positive hematopoietic progenitor cells for transplantation. J Hematother. 1995;4(6):515–525.
- Giarratana M-C, Kobari L, Tman N, et al. Cell culture bags allow a large extent of ex vivo expansion of LTC-IC and functional mature cells which can subsequently be frozen: interest for large-scale clinical applications. Bone Marrow Transplant. 1998;22(7):707–715.
- Traver D. Going with the flow: how shear stress signals the emergence of adult hematopoiesis. J Exp Med. 2015;212(5):600.
- Kowalczyk M, Waldron K, Kresnowati P, et al. Process challenges relating to hematopoietic stem cell cultivation in bioreactors. J Ind Microbiol Biotechnol. 2011;38(7):761–767.
- Nielsen LK. Bioreactors for hematopoietic cell culture. Annu Rev Biomed Eng. 1999;1(1):129–152.
- Madlambayan GJ, Rogers I, Purpura KA, et al. Clinically relevant expansion of hematopoietic stem cells with conserved function in a single-use, closed-system bioprocess. Biol Blood Marrow Transplant. 2006;12(10):1020–1030.
- Nicoud IB, Waters T, Blake J, et al. Integrated automation of multiple unit operations for processing and expanding cord blood hematopoietic stem and progenitor cells. Cytotherapy. 2015;17(6):S11.
- Parmar K, Mauch P, Vergilio J-A, et al. Distribution of hematopoietic stem cells in the bone marrow according to regional hypoxia. Proc Natl Acad Sci. 2007;104(13):5431–5436.
- Mohyeldin A, Garzón-Muvdi T, Quiñones-Hinojosa A. Oxygen in stem cell biology: a critical component of the stem cell niche. Cell Stem Cell. 2010;7(2):150–161.
- Kobayashi H, Morikawa T, Okinaga A, et al. Environmental optimization enables maintenance of quiescent hematopoietic stem cells ex vivo. Cell Rep. 2019;28(1):145–158.e9.
- Kumar S, Geiger H. HSC niche biology and HSC expansion ex vivo. Trends Mol Med. 2017;23(9):799–819.
- Li A, Kusuma GD, Driscoll D, et al. Advances in automated cell washing and concentration. Cytotherapy. 2021;23(9):774–786.
- Awan M, Buriak I, Fleck R, et al. Dimethyl sulfoxide: a central player since the Dawn of cryobiology, is efficacy balanced by toxicity? Regen Med. 2020;15(3):1463–1491.
- Hornberger K, Yu G, McKenna D, et al. Cryopreservation of hematopoietic stem cells: emerging assays, cryoprotectant agents, and technology to improve outcomes. Transfus Med Hemother. 2019;46(3):188–196.
- Berz D, McCormack EM, Winer ES, et al. Cryopreservation of hematopoietic stem cells. Am J Hematol. 2007;82(6):463–472.
- Hunt CJ. Technical considerations in the freezing, low-temperature storage and thawing of stem cells for cellular therapies. Transfus Med Hemother. 2019;46(3):134–150.
- Simon CG, Lin-Gibson S, Elliott JT, et al. Strategies for achieving measurement assurance for cell therapy products. Stem Cells Transl Med. 2016;5(6):705–708.
- Lin-Gibson S, Sarkar S, Elliott J, et al. Understanding and managing sources of variability in cell measurements. Cell Gene Ther Insights. 2016;2(6):663–673.
- Vembadi A, Menachery A, Qasaimeh MA. Cell cytometry: review and perspective on biotechnological advances. Front Bioeng Biotechnol. 2019;7:147.
- der Strate B, Van, Longdin R, Geerlings M, et al. Best practices in performing flow cytometry in a regulated environment: feedback from experience within the European bioanalysis forum. Bioanalysis. 2017;9(16):1253–1264.
- Wood B, Jevremovic D, Béné MC, et al. Validation of cell-based fluorescence assays: practice guidelines from the ICSH and ICCS - part V - assay performance criteria: assay performance criteria for cell-based fluorescence assays. Cytometry B Clin Cytom. 2013;84(5):315–323.
- Skific M, Golemovic M. Colony-forming unit assay as a potency test for hematopoietic stem/progenitor cell products. Mol Exp Biol Med. 2019;2(2):51–56.
- Lin H-T, Okumura T, Yatsuda Y, et al. Application of droplet digital PCR for estimating vector copy number states in stem cell gene therapy. Hum Gene Ther Methods. 2016;27(5):197–208.
- Santeramo I, Bagnati M, Harvey EJ, et al. Vector copy distribution at a single-cell level enhances analytical characterization of gene-modified cell therapies. Mol Ther - Methods Clin Dev. 2020;17:944–956.
- Findlay SD, Vincent KM, Berman JR, et al. A Digital PCR-Based Method for Efficient and Highly Specific Screening of Genome Edited Cells. Wang TT, editor. PLOS ONE. 2016;11:e0153901.
- Sentmanat MF, Peters ST, Florian CP, et al. A survey of validation strategies for CRISPR-Cas9 editing. Sci Rep. 2018;8(1):888.
- Yang Z, Steentoft C, Hauge C, et al. Fast and sensitive detection of indels induced by precise gene targeting. Nucleic Acids Res. 2015;43(9):e59–e59.
- Brinkman EK, Chen T, Amendola M, et al. Easy quantitative assessment of genome editing by sequence trace decomposition. Nucleic Acids Res. 2014;42(22):e168–e168.
- Bennett EP, Petersen BL, Johansen IE, et al. INDEL detection, the ‘Achilles heel’ of precise genome editing: a survey of methods for accurate profiling of gene editing induced indels. Nucleic Acids Res. 2020;48(21):11958–11981.
- England MR, Stock F, Gebo JET, et al. Comprehensive Evaluation of Compendial USP, BacT/Alert Dual-T, and Bactec FX for Detection of Product Sterility Testing Contaminants. Munson E, editor. J Clin Microbiol. 2019;57:e01548–18.
- Gee AP, Sumstad D, Stanson J, et al. A multicenter comparison study between the Endosafe® PTSTM rapid-release testing system and traditional methods for detecting endotoxin in cell-therapy products. Cytotherapy. 2008;10(4):427–435.
- D’Apolito D, D’Aiello L, Pasqua S, et al. Strategy and validation of a consistent and reproducible nucleic acid technique for mycoplasma detection in advanced therapy medicinal products. Biologicals. 2020;64:49–57.
- World Health Organization. WHO/BS/2019.2373 Proposed WHO 1st Inter Reference Panel (19/158) for Quantitation of Lentiviral Vector Integration Copy Numbers [Internet]. [cited 2022 Jun 19]. Available from: https://www.who.int/publications/m/item/WHO-BS-2019.2373.
- Blattner G, Cavazza A, Thrasher AJ, et al. Gene editing and genotoxicity: targeting the off-targets. Front Genome Ed. 2020;2:613252.
- Seita J, Weissman IL. Hematopoietic stem cell: self‐renewal versus differentiation. WIREs Syst Biol Med. 2010;2(6):640–653.
- Dao M, Nolta J. CD34: to select or not to select? That is the question. Leukemia. 2000;14(5):773–776.
- FDA. Studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial [Internet]. [cited 2022 Jun 19]. Available from: https://www.fda.gov/media/152536/download.
- Li C, Georgakopoulou A, Mishra A, et al. In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice. Blood Adv. 2021;5(4):1122–1135.