{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,30]],"date-time":"2026-03-30T08:00:54Z","timestamp":1774857654114,"version":"3.50.1"},"reference-count":102,"publisher":"Springer Science and Business Media LLC","issue":"3","license":[{"start":{"date-parts":[[2025,4,25]],"date-time":"2025-04-25T00:00:00Z","timestamp":1745539200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,4,25]],"date-time":"2025-04-25T00:00:00Z","timestamp":1745539200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"funder":[{"DOI":"10.13039\/501100006407","name":"Natural Science Foundation of Henan Province","doi-asserted-by":"crossref","award":["242300421199"],"award-info":[{"award-number":["242300421199"]}],"id":[{"id":"10.13039\/501100006407","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100006407","name":"Natural Science Foundation of Henan Province","doi-asserted-by":"publisher","award":["242300420117"],"award-info":[{"award-number":["242300420117"]}],"id":[{"id":"10.13039\/501100006407","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Henan Province Joint Fund for Science and Technology Research and Development","award":["235101610002"],"award-info":[{"award-number":["235101610002"]}]},{"name":"Key Technologies R&D Program of Henan Province","award":["242102310134"],"award-info":[{"award-number":["242102310134"]}]},{"DOI":"10.13039\/501100017664","name":"Henan Province Foundation for University Key Teacher","doi-asserted-by":"publisher","award":["2024GGJS088"],"award-info":[{"award-number":["2024GGJS088"]}],"id":[{"id":"10.13039\/501100017664","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Key Scientific Research Program of Higher Education in Henan Province","award":["24A180026"],"award-info":[{"award-number":["24A180026"]}]},{"name":"Key Scientific Research Program of Higher Education in Henan Province","award":["23A180005"],"award-info":[{"award-number":["23A180005"]}]},{"name":"Research Innovation Support Program for Postgraduates of Xinxiang Medical University","award":["YJSCX202360Y"],"award-info":[{"award-number":["YJSCX202360Y"]}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Mol Biotechnol"],"published-print":{"date-parts":[[2026,3]]},"DOI":"10.1007\/s12033-025-01435-3","type":"journal-article","created":{"date-parts":[[2025,4,25]],"date-time":"2025-04-25T13:15:15Z","timestamp":1745586915000},"page":"1068-1078","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Therapeutic Potential of Mesenchymal Stem Cells in Niemann\u2013Pick Disease"],"prefix":"10.1007","volume":"68","author":[{"given":"Keli","family":"Xu","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Minlin","family":"Yang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lihong","family":"Guan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ciqing","family":"Yang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Liang","family":"Qiao","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yonghai","family":"Li","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4203-8117","authenticated-orcid":false,"given":"Juntang","family":"Lin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiaoying","family":"Li","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2025,4,25]]},"reference":[{"issue":"4","key":"1435_CR1","doi-asserted-by":"publisher","DOI":"10.1002\/cbf.4028","volume":"42","author":"K Hosseini","year":"2024","unstructured":"Hosseini, K., Fallahi, J., Razban, V., Sirat, R. Z., Varasteh, M., & Tarhriz, V. (2024). Overview of clinical, molecular, and therapeutic features of Niemann\u2013Pick disease (types A, B, and C): Focus on therapeutic approaches. Cell Biochemistry and Function, 42(4), e4028.","journal-title":"Cell Biochemistry and Function"},{"key":"1435_CR2","doi-asserted-by":"publisher","first-page":"1717","DOI":"10.1016\/B978-0-444-59565-2.00041-1","volume":"113","author":"MT Vanier","year":"2013","unstructured":"Vanier, M. T. (2013). Niemann\u2013Pick diseases. Handbook of Clinical Neurology, 113, 1717\u20131721.","journal-title":"Handbook of Clinical Neurology"},{"issue":"1","key":"1435_CR3","doi-asserted-by":"publisher","first-page":"16","DOI":"10.1016\/j.ymgme.2020.02.002","volume":"130","author":"ECB Eskes","year":"2020","unstructured":"Eskes, E. C. B., Sjouke, B., Vaz, F. M., Goorden, S. M. I., van Kuilenburg, A. B. P., Aerts, J. M. F. G., & Hollak, C. E. M. (2020). Biochemical and imaging parameters in acid sphingomyelinase deficiency: Potential utility as biomarkers. Molecular Genetics and Metabolism, 130(1), 16\u201326.","journal-title":"Molecular Genetics and Metabolism"},{"key":"1435_CR4","doi-asserted-by":"publisher","first-page":"1002574","DOI":"10.3389\/fnut.2022.1002574","volume":"9","author":"F Yang","year":"2022","unstructured":"Yang, F., & Chen, G. (2022). The nutritional functions of dietary sphingomyelin and its applications in food. Frontiers in Nutrition, 9, 1002574.","journal-title":"Frontiers in Nutrition"},{"issue":"2","key":"1435_CR5","doi-asserted-by":"publisher","first-page":"60","DOI":"10.1111\/j.1751-2980.2011.00481.x","volume":"12","author":"RD Duan","year":"2011","unstructured":"Duan, R. D. (2011). Physiological functions and clinical implications of sphingolipids in the gut. Journal of Digestive Diseases, 12(2), 60\u201370.","journal-title":"Journal of Digestive Diseases"},{"issue":"1","key":"1435_CR6","doi-asserted-by":"publisher","first-page":"71","DOI":"10.1080\/09687860500445604","volume":"23","author":"DE Michael","year":"2009","unstructured":"Michael, D. E., & Hansen, G. H. (2009). Lipid raft organization and function in brush borders of epithelial cells (Review). Molecular Membrane Biology, 23(1), 71\u201379.","journal-title":"Molecular Membrane Biology"},{"issue":"1","key":"1435_CR7","doi-asserted-by":"publisher","first-page":"8259","DOI":"10.1038\/s41598-021-87795-7","volume":"11","author":"F Al-Rashed","year":"2021","unstructured":"Al-Rashed, F., Ahmad, Z., Snider, A. J., Thomas, R., Kochumon, S., Melhem, M., & Ahmad, R. (2021). Ceramide kinase regulates TNF-\u03b1-induced immune responses in human monocytic cells. Scientific Reports, 11(1), 8259.","journal-title":"Scientific Reports"},{"issue":"4","key":"1435_CR8","doi-asserted-by":"publisher","first-page":"1067","DOI":"10.1016\/j.jaci.2022.12.808","volume":"151","author":"K Yoshida","year":"2023","unstructured":"Yoshida, K., Morishima, Y., Ano, S., Sakurai, H., Kuramoto, K., Tsunoda, Y., & Hizawa, N. (2023). ELOVL6 deficiency aggravates allergic airway inflammation through the ceramide-S1P pathway in mice. Journal of Allergy and Clinical Immunology, 151(4), 1067\u20131080.","journal-title":"Journal of Allergy and Clinical Immunology"},{"key":"1435_CR9","doi-asserted-by":"publisher","first-page":"4806541","DOI":"10.1155\/2017\/4806541","volume":"2017","author":"NC Hait","year":"2017","unstructured":"Hait, N. C., & Maiti, A. (2017). The role of sphingosine-1-phosphate and ceramide-1-phosphate in inflammation and cancer. Mediators of Inflammation, 2017, 4806541.","journal-title":"Mediators of Inflammation"},{"issue":"5","key":"1435_CR10","doi-asserted-by":"publisher","first-page":"654","DOI":"10.1007\/s10545-007-0632-9","volume":"30","author":"EH Schuchman","year":"2007","unstructured":"Schuchman, E. H. (2007). The pathogenesis and treatment of acid sphingomyelinase-deficient Niemann\u2013Pick disease. Journal of Inherited Metabolic Disease, 30(5), 654\u2013663.","journal-title":"Journal of Inherited Metabolic Disease"},{"issue":"1","key":"1435_CR11","doi-asserted-by":"publisher","first-page":"27","DOI":"10.1016\/j.ymgme.2016.12.008","volume":"120","author":"EH Schuchman","year":"2017","unstructured":"Schuchman, E. H., & Desnick, R. J. (2017). Types A and B Niemann\u2013Pick disease. Molecular Genetics and Metabolism, 120(1), 27\u201333.","journal-title":"Molecular Genetics and Metabolism"},{"issue":"2","key":"1435_CR12","doi-asserted-by":"publisher","first-page":"211","DOI":"10.3390\/biom14020211","volume":"14","author":"C Tirelli","year":"2024","unstructured":"Tirelli, C., Rondinone, O., Italia, M., Mira, S., Belmonte, L. A., De Grassi, M., & Centanni, S. (2024). The genetic basis, lung involvement, and therapeutic options in Niemann\u2013Pick disease: A comprehensive review. Biomolecules, 14(2), 211.","journal-title":"Biomolecules"},{"issue":"4","key":"1435_CR13","doi-asserted-by":"publisher","first-page":"466","DOI":"10.1016\/j.ymgme.2019.02.004","volume":"126","author":"SE Bianconi","year":"2019","unstructured":"Bianconi, S. E., Hammond, D. I., Farhat, N. Y., Dang, D. A., Jenkins, K., Cougnoux, A., & Porter, F. D. (2019). Evaluation of age of death in Niemann\u2013Pick disease, type C: Utility of disease support group websites to understand natural history. Molecular Genetics and Metabolism, 126(4), 466\u2013469.","journal-title":"Molecular Genetics and Metabolism"},{"issue":"1","key":"1435_CR14","doi-asserted-by":"publisher","first-page":"132","DOI":"10.1002\/ajmg.b.30969","volume":"153","author":"NM Yanjanin","year":"2009","unstructured":"Yanjanin, N. M., V\u00e9lez, J. I., Gropman, A., King, K., Bianconi, S. E., Conley, S. K., & Porter, F. D. (2009). Linear clinical progression, independent of age of onset, in Niemann\u2013Pick disease, type C. American Journal of Medical Genetics Part B: Neuropsychiatric Genetics, 153(1), 132\u2013140.","journal-title":"American Journal of Medical Genetics Part B: Neuropsychiatric Genetics"},{"issue":"1","key":"1435_CR15","doi-asserted-by":"publisher","first-page":"120","DOI":"10.1093\/brain\/awl260","volume":"130","author":"M Sevin","year":"2006","unstructured":"Sevin, M., Lesca, G., Baumann, N., Millat, G., Lyon-Caen, O., Vanier, M. T., & Sedel, F. (2006). The adult form of Niemann\u2013Pick disease type C. Brain, 130(1), 120\u2013133.","journal-title":"Brain"},{"issue":"11","key":"1435_CR16","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1038\/s12276-018-0176-0","volume":"50","author":"I Kang","year":"2018","unstructured":"Kang, I., Lee, B. C., Lee, J. Y., Kim, J.-J., Sung, E.-A., Lee, S. E., & Kang, K.-S. (2018). Stem cell-secreted 14, 15-epoxyeicosatrienoic acid rescues cholesterol homeostasis and autophagic flux in Niemann\u2013Pick-type C disease. Experimental and Molecular Medicine, 50(11), 1\u201314.","journal-title":"Experimental and Molecular Medicine"},{"issue":"1","key":"1435_CR17","doi-asserted-by":"publisher","first-page":"50","DOI":"10.1186\/s13023-018-0785-7","volume":"13","author":"T Geberhiwot","year":"2018","unstructured":"Geberhiwot, T., Moro, A., Dardis, A., Ramaswami, U., Sirrs, S., Marfa, M. P., & Patterson, M. (2018). Consensus clinical management guidelines for Niemann\u2013Pick disease type C. Orphanet Journal of Rare Diseases, 13(1), 50.","journal-title":"Orphanet Journal of Rare Diseases"},{"key":"1435_CR18","doi-asserted-by":"publisher","first-page":"139","DOI":"10.1007\/978-3-030-14265-0_8","volume":"1135","author":"SM Cologna","year":"2019","unstructured":"Cologna, S. M., & Rosenhouse-Dantsker, A. (2019). Insights into the molecular mechanisms of cholesterol binding to the NPC1 and NPC2 proteins. Advances in Experimental Medicine and Biology, 1135, 139\u2013160.","journal-title":"Advances in Experimental Medicine and Biology"},{"key":"1435_CR19","doi-asserted-by":"publisher","first-page":"16","DOI":"10.1186\/1750-1172-5-16","volume":"5","author":"MT Vanier","year":"2010","unstructured":"Vanier, M. T. (2010). Niemann\u2013Pick disease type C. Orphanet Journal of Rare Diseases, 5, 16.","journal-title":"Orphanet Journal of Rare Diseases"},{"issue":"11","key":"1435_CR20","doi-asserted-by":"publisher","first-page":"963","DOI":"10.3109\/00207454.2016.1161623","volume":"126","author":"V Di Lazzaro","year":"2016","unstructured":"Di Lazzaro, V., Marano, M., Florio, L., & De Santis, S. (2016). Niemann\u2013Pick type C: Focus on the adolescent\/adult onset form. International Journal of Neuroscience, 126(11), 963\u2013971.","journal-title":"International Journal of Neuroscience"},{"issue":"4","key":"1435_CR21","doi-asserted-by":"publisher","first-page":"107","DOI":"10.1097\/YPG.0000000000000286","volume":"31","author":"LT Ong","year":"2021","unstructured":"Ong, L. T. (2021). Psychosis symptoms associated with Niemann\u2013Pick disease type C. Psychiatric Genetics, 31(4), 107\u2013118.","journal-title":"Psychiatric Genetics"},{"issue":"1","key":"1435_CR22","doi-asserted-by":"publisher","first-page":"51","DOI":"10.1186\/s13023-022-02200-4","volume":"17","author":"SC Bolton","year":"2022","unstructured":"Bolton, S. C., Soran, V., Marfa, M. P., Imrie, J., Gissen, P., Jahnova, H., & Geberhiwot, T. (2022). Clinical disease characteristics of patients with Niemann\u2013Pick Disease Type C: Findings from the International Niemann\u2013Pick Disease Registry (INPDR). Orphanet Journal of Rare Diseases, 17(1), 51.","journal-title":"Orphanet Journal of Rare Diseases"},{"issue":"8","key":"1435_CR23","doi-asserted-by":"publisher","first-page":"1006","DOI":"10.1089\/hum.2005.16.1006","volume":"16","author":"J-S Bae","year":"2005","unstructured":"Bae, J.-S., Furuya, S., Shinoda, Y., Endo, S., Schuchman, E. H., Hirabayashi, Y., & Jin, H. K. (2005). Neurodegeneration augments the ability of bone marrow-derived mesenchymal stem cells to fuse with Purkinje neurons in Niemann\u2013Pick type C mice. Human Gene Therapy, 16(8), 1006\u20131011.","journal-title":"Human Gene Therapy"},{"issue":"14","key":"1435_CR24","doi-asserted-by":"publisher","first-page":"5059","DOI":"10.3390\/ijms21145059","volume":"21","author":"YB Seker","year":"2020","unstructured":"Seker, Y. B., Baruteau, J., Rahim, A. A., & Gissen, P. (2020). Clinical and molecular features of early infantile Niemann Pick Type C disease. International Journal of Molecular Sciences, 21(14), 5059.","journal-title":"International Journal of Molecular Sciences"},{"issue":"1","key":"1435_CR25","doi-asserted-by":"publisher","first-page":"139","DOI":"10.1086\/513899","volume":"61","author":"WL Greer","year":"1997","unstructured":"Greer, W. L., Riddell, D. C., Byers, D. M., Welch, J. P., Girouard, G. S., Sparrow, S. M., & Neumann, P. E. (1997). Linkage of Niemann\u2013Pick disease type D to the same region of human chromosome 18 as Niemann\u2013Pick disease type C. American Journal of Human Genetics, 61(1), 139\u2013142.","journal-title":"American Journal of Human Genetics"},{"key":"1435_CR26","unstructured":"Bajwa, H., & Azhar, W. (2023). Niemann\u2013Pick disease. In S. Aboubakr, A. Abu-Ghosh, W. B. Ackley, P. Adibi Sedeh, T. S. Adolphe, T. C. Aeby, N. R. Aeddula, S. Agadi, P. Agasthi, A. Ahmed, & F. W. Ahmed (Eds.), StatPearls. StatPearls Publishing."},{"issue":"22","key":"1435_CR27","first-page":"360","volume":"3","author":"A Santos-Lozano","year":"2015","unstructured":"Santos-Lozano, A., Villamandos, G. D., Sanchis-Gomar, F., Fiuza-Luces, C., Pareja-Galeano, H., Garatachea, N., & Lucia, A. (2015). Niemann\u2013Pick disease treatment: A systematic review of clinical trials. Annals of Translational Medicine, 3(22), 360.","journal-title":"Annals of Translational Medicine"},{"issue":"9214","key":"1435_CR28","doi-asserted-by":"publisher","first-page":"1481","DOI":"10.1016\/S0140-6736(00)02161-9","volume":"355","author":"T Cox","year":"2000","unstructured":"Cox, T., Lachmann, R., Hollak, C., Aerts, J., van Weely, S., Hreb\u00edcek, M., & Zimran, A. (2000). Novel oral treatment of Gaucher\u2019s disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Lancet, 355(9214), 1481\u20131485.","journal-title":"Lancet"},{"issue":"3","key":"1435_CR29","doi-asserted-by":"publisher","first-page":"330","DOI":"10.1016\/j.ymgme.2012.03.012","volume":"106","author":"MC Patterson","year":"2012","unstructured":"Patterson, M. C., Hendriksz, C. J., Walterfang, M., Sedel, F., Vanier, M. T., & Wijburg, F. (2012). Recommendations for the diagnosis and management of Niemann\u2013Pick disease type C: An update. Molecular Genetics and Metabolism, 106(3), 330\u2013344.","journal-title":"Molecular Genetics and Metabolism"},{"issue":"9","key":"1435_CR30","doi-asserted-by":"publisher","first-page":"765","DOI":"10.1016\/S1474-4422(07)70194-1","volume":"6","author":"MC Patterson","year":"2007","unstructured":"Patterson, M. C., Vecchio, D., Prady, H., Abel, L., & Wraith, J. E. (2007). Miglustat for treatment of Niemann\u2013Pick C disease: A randomised controlled study. The Lancet Neurology, 6(9), 765\u2013772.","journal-title":"The Lancet Neurology"},{"issue":"3","key":"1435_CR31","doi-asserted-by":"publisher","first-page":"243","DOI":"10.1016\/j.ymgme.2009.07.003","volume":"98","author":"M Pineda","year":"2009","unstructured":"Pineda, M., Wraith, J. E., Mengel, E., Sedel, F., Hwu, W. L., Rohrbach, M., & Patterson, M. C. (2009). Miglustat in patients with Niemann\u2013Pick disease Type C (NP-C): A multicenter observational retrospective cohort study. Molecular Genetics and Metabolism, 98(3), 243\u2013249.","journal-title":"Molecular Genetics and Metabolism"},{"issue":"3","key":"1435_CR32","doi-asserted-by":"publisher","first-page":"300","DOI":"10.1177\/0883073809344222","volume":"25","author":"MC Patterson","year":"2010","unstructured":"Patterson, M. C., Vecchio, D., Jacklin, E., Abel, L., Chadha-Boreham, H., Luzy, C., & Wraith, J. E. (2010). Long-term miglustat therapy in children with Niemann\u2013Pick disease type C. Journal of Child Neurology, 25(3), 300\u2013305.","journal-title":"Journal of Child Neurology"},{"issue":"4","key":"1435_CR33","doi-asserted-by":"publisher","first-page":"351","DOI":"10.1016\/j.ymgme.2009.12.006","volume":"99","author":"JE Wraith","year":"2010","unstructured":"Wraith, J. E., Vecchio, D., Jacklin, E., Abel, L., Chadha-Boreham, H., Luzy, C., & Patterson, M. C. (2010). Miglustat in adult and juvenile patients with Niemann\u2013Pick disease type C: Long-term data from a clinical trial. Molecular Genetics and Metabolism, 99(4), 351\u2013357.","journal-title":"Molecular Genetics and Metabolism"},{"key":"1435_CR34","doi-asserted-by":"publisher","DOI":"10.1002\/trc2.70029","author":"M Jan","year":"2025","unstructured":"Jan, M., Akbar, H. M., Ashfaque, M., Khan, M. L., Talha, M., & Haque, M. A. (2025). FDA approval of Miplyffa and Aqneursa: A dual breakthrough for the treatment of Neimann-Pick disease type C. Alzheimer\u2019s and Dementia: Translational Research and Clinical Interventions. https:\/\/doi.org\/10.1002\/trc2.70029","journal-title":"Alzheimer's and Dementia: Translational Research and Clinical Interventions"},{"issue":"12","key":"1435_CR35","doi-asserted-by":"publisher","first-page":"1089","DOI":"10.1016\/j.clinthera.2024.10.010","volume":"46","author":"P Beninger","year":"2024","unstructured":"Beninger, P. (2024). Miplyffa (arimoclomol). Clinical Therapeutics, 46(12), 1089\u20131090.","journal-title":"Clinical Therapeutics"},{"issue":"8","key":"1435_CR36","doi-asserted-by":"publisher","first-page":"2215","DOI":"10.1007\/s11011-021-00842-0","volume":"36","author":"D Sitarska","year":"2021","unstructured":"Sitarska, D., Tylki-Szyma\u0144ska, A., & \u0141ugowska, A. (2021). Treatment trials in Niemann\u2013Pick type C disease. Metabolic Brain Disease, 36(8), 2215\u20132221.","journal-title":"Metabolic Brain Disease"},{"issue":"2","key":"1435_CR37","doi-asserted-by":"publisher","first-page":"131","DOI":"10.1016\/S0024-3205(01)01384-4","volume":"70","author":"F Camargo","year":"2001","unstructured":"Camargo, F., Erickson, R. P., Garver, W. S., Hossain, G. S., Carbone, P. N., Heidenreich, R. A., & Blanchard, J. (2001). Cyclodextrins in the treatment of a mouse model of Niemann\u2013Pick C disease. Life Science, 70(2), 131\u2013142.","journal-title":"Life Science"},{"issue":"7","key":"1435_CR38","doi-asserted-by":"publisher","first-page":"2377","DOI":"10.1073\/pnas.0810895106","volume":"106","author":"B Liu","year":"2009","unstructured":"Liu, B., Turley, S. D., Burns, D. K., Miller, A. M., Repa, J. J., & Dietschy, J. M. (2009). Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1\u2212\/\u2212 mouse. Proceedings of the National Academy of Sciences of USA, 106(7), 2377\u20132382.","journal-title":"Proceedings of the National Academy of Sciences of USA"},{"issue":"9","key":"1435_CR39","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0006951","volume":"4","author":"CD Davidson","year":"2009","unstructured":"Davidson, C. D., Ali, N. F., Micsenyi, M. C., Stephney, G., Renault, S., Dobrenis, K., & Walkley, S. U. (2009). Chronic cyclodextrin treatment of murine Niemann\u2013Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression. PLoS ONE, 4(9), e6951.","journal-title":"PLoS ONE"},{"issue":"276","key":"1435_CR40","doi-asserted-by":"publisher","first-page":"276ra226","DOI":"10.1126\/scitranslmed.3010101","volume":"7","author":"CH Vite","year":"2015","unstructured":"Vite, C. H., Bagel, J. H., Swain, G. P., Prociuk, M., Sikora, T. U., Stein, V. M., & Walkley, S. U. (2015). Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann\u2013Pick type C1 disease. Science Translational Medicine, 7(276), 276ra226.","journal-title":"Science Translational Medicine"},{"key":"1435_CR41","doi-asserted-by":"crossref","unstructured":"Mylvara, A. V., Gibson, A. L., Gu, T., Davidson, C. D., Incao, A. A., Melnyk, K., & Pavan, W. J. (2024). Optimization of systemic AAV9 gene therapy in Niemann\u2013Pick disease type C1 mice. bioRxiv.","DOI":"10.1101\/2024.06.07.597901"},{"issue":"10","key":"1435_CR42","doi-asserted-by":"publisher","DOI":"10.26508\/lsa.202101040","volume":"4","author":"CD Davidson","year":"2021","unstructured":"Davidson, C. D., Gibson, A. L., Gu, T., Baxter, L. L., Deverman, B. E., Beadle, K., & Pavan, W. J. (2021). Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann\u2013Pick disease type C1 mice. Life Science Alliance, 4(10), e202101040.","journal-title":"Life Science Alliance"},{"issue":"12","key":"1435_CR43","doi-asserted-by":"publisher","first-page":"1619","DOI":"10.3390\/cells12121619","volume":"12","author":"MP Hughes","year":"2023","unstructured":"Hughes, M. P., Nelvagal, H. R., Coombe-Tennant, O., Smith, D., Smith, C., Massaro, G., & Rahim, A. A. (2023). A novel small NPC1 promoter enhances AAV-mediated gene therapy in mouse models of Niemann\u2013Pick type C1 disease. Cells, 12(12), 1619.","journal-title":"Cells"},{"issue":"17","key":"1435_CR44","doi-asserted-by":"publisher","first-page":"3079","DOI":"10.1093\/hmg\/ddy212","volume":"27","author":"MP Hughes","year":"2018","unstructured":"Hughes, M. P., Smith, D. A., Morris, L., Fletcher, C., Colaco, A., Huebecker, M., & Rahim, A. A. (2018). AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann\u2013Pick type C1 disease. Human Molecular Genetics, 27(17), 3079\u20133098.","journal-title":"Human Molecular Genetics"},{"key":"1435_CR45","doi-asserted-by":"publisher","first-page":"22","DOI":"10.1016\/j.expneurol.2018.04.001","volume":"306","author":"S Markmann","year":"2018","unstructured":"Markmann, S., Christie-Reid, J. J., Rosenberg, J. B., De, B. P., Kaminsky, S. M., Crystal, R. G., & Sondhi, D. (2018). Attenuation of the Niemann\u2013Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2. Experimental Neurology, 306, 22\u201333.","journal-title":"Experimental Neurology"},{"issue":"11\u201312","key":"1435_CR46","doi-asserted-by":"publisher","first-page":"589","DOI":"10.1089\/hum.2020.175","volume":"32","author":"Y Kurokawa","year":"2021","unstructured":"Kurokawa, Y., Osaka, H., Kouga, T., Jimbo, E., Muramatsu, K., Nakamura, S., & Yamagata, T. (2021). Gene therapy in a mouse model of Niemann\u2013Pick disease type C1. Human Gene Therapy, 32(11\u201312), 589\u2013598.","journal-title":"Human Gene Therapy"},{"issue":"S2","key":"1435_CR47","doi-asserted-by":"publisher","first-page":"120","DOI":"10.1186\/s13052-018-0562-1","volume":"44","author":"D Concolino","year":"2018","unstructured":"Concolino, D., Deodato, F., & Parini, R. (2018). Enzyme replacement therapy: Efficacy and limitations. Italian Journal of Pediatrics, 44(S2), 120.","journal-title":"Italian Journal of Pediatrics"},{"issue":"13","key":"1435_CR48","doi-asserted-by":"publisher","first-page":"1988","DOI":"10.1096\/fj.00-0014com","volume":"14","author":"SR Miranda","year":"2000","unstructured":"Miranda, S. R., He, X., Simonaro, C. M., Gatt, S., Dagan, A., Desnick, R. J., & Schuchman, E. H. (2000). Infusion of recombinant human acid sphingomyelinase into Niemann\u2013Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology. FASEB Journal, 14(13), 1988\u20131995.","journal-title":"FASEB Journal"},{"issue":"8","key":"1435_CR49","doi-asserted-by":"publisher","first-page":"941","DOI":"10.1007\/s40265-022-01727-x","volume":"82","author":"SJ Keam","year":"2022","unstructured":"Keam, S. J. (2022). Olipudase alfa: First approval. Drugs, 82(8), 941\u2013947.","journal-title":"Drugs"},{"key":"1435_CR50","doi-asserted-by":"publisher","DOI":"10.1016\/j.gimo.2024.101888","volume":"2","author":"M Kumar","year":"2024","unstructured":"Kumar, M., Aguiar, M., Jessel, A., Thurberg, B. L., Underhill, L., Wong, H., & Schuchman, E. H. (2024). The impact of sphingomyelin on the pathophysiology and treatment response to olipudase alfa in acid sphingomyelinase deficiency. Genetics in Medicine Open, 2, 101888.","journal-title":"Genetics in Medicine Open"},{"issue":"1","key":"1435_CR51","doi-asserted-by":"publisher","first-page":"94","DOI":"10.1186\/s13023-023-02700-x","volume":"18","author":"RH Lachmann","year":"2023","unstructured":"Lachmann, R. H., Diaz, G. A., Wasserstein, M. P., Armstrong, N. M., Yarramaneni, A., Kim, Y., & Kumar, M. (2023). Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): Sustained improvements in clinical outcomes after 6.5 years of treatment in adults. Orphanet Journal of Rare Diseases, 18(1), 94.","journal-title":"Orphanet Journal of Rare Diseases"},{"issue":"5","key":"1435_CR52","doi-asserted-by":"publisher","first-page":"369","DOI":"10.1007\/s40261-023-01270-x","volume":"43","author":"YY Syed","year":"2023","unstructured":"Syed, Y. Y. (2023). Olipudase alfa in non-CNS manifestations of acid sphingomyelinase deficiency: A profile of its use. Clinical Drug Investigation, 43(5), 369\u2013377.","journal-title":"Clinical Drug Investigation"},{"issue":"7","key":"1435_CR53","doi-asserted-by":"publisher","first-page":"801","DOI":"10.1177\/0963689719837897","volume":"28","author":"A Musia\u0142-Wysocka","year":"2019","unstructured":"Musia\u0142-Wysocka, A., Kot, M., & Majka, M. (2019). The pros and cons of mesenchymal stem cell-based therapies. Cell Transplantation, 28(7), 801\u2013812.","journal-title":"Cell Transplantation"},{"issue":"9","key":"1435_CR54","doi-asserted-by":"publisher","first-page":"779","DOI":"10.3390\/cells13090779","volume":"13","author":"V Mattei","year":"2024","unstructured":"Mattei, V., & Delle, M. S. (2024). Mesenchymal stem cells and their role in neurodegenerative diseases. Cells, 13(9), 779.","journal-title":"Cells"},{"issue":"3","key":"1435_CR55","doi-asserted-by":"publisher","first-page":"330","DOI":"10.1001\/jama.2016.17822","volume":"317","author":"J Jin","year":"2017","unstructured":"Jin, J. (2017). Stem cell treatments. JAMA, 317(3), 330.","journal-title":"JAMA"},{"issue":"5","key":"1435_CR56","doi-asserted-by":"publisher","first-page":"785","DOI":"10.3892\/ol.2021.13046","volume":"22","author":"S Lim","year":"2021","unstructured":"Lim, S., & Khoo, B. (2021). An overview of mesenchymal stem cells and their potential therapeutic benefits in cancer therapy (Review). Oncology Letters, 22(5), 785.","journal-title":"Oncology Letters"},{"issue":"1","key":"1435_CR57","doi-asserted-by":"publisher","first-page":"135","DOI":"10.1186\/s13287-017-0584-4","volume":"8","author":"S Sun","year":"2017","unstructured":"Sun, S., Li, Z., Glencer, P., Cai, B., Zhang, X., Yang, J., & Li, X. (2017). Bringing the age-related macular degeneration high-risk allele age-related maculopathy susceptibility 2 into focus with stem cell technology. Stem Cell Research and Therapy, 8(1), 135.","journal-title":"Stem Cell Research and Therapy"},{"issue":"5","key":"1435_CR58","doi-asserted-by":"publisher","first-page":"115","DOI":"10.1186\/scrt326","volume":"4","author":"J Liu","year":"2013","unstructured":"Liu, J. (2013). Induced pluripotent stem cell-derived neural stem cells: New hope for stroke? Stem Cell Research and Therapy, 4(5), 115.","journal-title":"Stem Cell Research and Therapy"},{"issue":"5","key":"1435_CR59","doi-asserted-by":"publisher","first-page":"1005","DOI":"10.1016\/j.bone.2011.07.032","volume":"49","author":"V Gangji","year":"2011","unstructured":"Gangji, V., De Maertelaer, V., & Hauzeur, J. P. (2011). Autologous bone marrow cell implantation in the treatment of non-traumatic osteonecrosis of the femoral head: Five year follow-up of a prospective controlled study. Bone, 49(5), 1005\u20131009.","journal-title":"Bone"},{"issue":"1","key":"1435_CR60","doi-asserted-by":"publisher","first-page":"325","DOI":"10.1016\/j.bone.2011.11.002","volume":"50","author":"D Zhao","year":"2012","unstructured":"Zhao, D., Cui, D., Wang, B., Tian, F., Guo, L., Yang, L., & Yu, X. (2012). Treatment of early stage osteonecrosis of the femoral head with autologous implantation of bone marrow-derived and cultured mesenchymal stem cells. Bone, 50(1), 325\u2013330.","journal-title":"Bone"},{"issue":"1","key":"1435_CR61","doi-asserted-by":"publisher","first-page":"355","DOI":"10.1186\/s13287-018-1099-3","volume":"9","author":"HM Shahjalal","year":"2018","unstructured":"Shahjalal, H. M., Abdal, D. A., Lim, K. M., Jeon, T. I., & Cho, S. G. (2018). Generation of pancreatic beta cells for treatment of diabetes: Advances and challenges. Stem Cell Research and Therapy, 9(1), 355.","journal-title":"Stem Cell Research and Therapy"},{"issue":"1","key":"1435_CR62","doi-asserted-by":"publisher","first-page":"111","DOI":"10.1186\/s13287-017-0567-5","volume":"8","author":"T Duncan","year":"2017","unstructured":"Duncan, T., & Valenzuela, M. (2017). Alzheimer\u2019s disease, dementia, and stem cell therapy. Stem Cell Research and Therapy, 8(1), 111.","journal-title":"Stem Cell Research and Therapy"},{"issue":"1","key":"1435_CR63","doi-asserted-by":"publisher","first-page":"371","DOI":"10.1186\/s13287-022-03050-4","volume":"13","author":"RM Heris","year":"2022","unstructured":"Heris, R. M., Shirvaliloo, M., Abbaspour-Aghdam, S., Hazrati, A., Shariati, A., Youshanlouei, H. R., & Ahmadi, M. (2022). The potential use of mesenchymal stem cells and their exosomes in Parkinson\u2019s disease treatment. Stem Cell Research and Therapy, 13(1), 371.","journal-title":"Stem Cell Research and Therapy"},{"key":"1435_CR64","doi-asserted-by":"publisher","first-page":"2853650","DOI":"10.1155\/2020\/2853650","volume":"2020","author":"L Gao","year":"2020","unstructured":"Gao, L., Peng, Y., Xu, W., He, P., Li, T., Lu, X., & Chen, G. (2020). Progress in stem cell therapy for spinal cord injury. Stem Cells International, 2020, 2853650.","journal-title":"Stem Cells International"},{"issue":"12","key":"1435_CR65","doi-asserted-by":"publisher","first-page":"3827","DOI":"10.1007\/s00167-020-05859-z","volume":"28","author":"EC Doyle","year":"2020","unstructured":"Doyle, E. C., Wragg, N. M., & Wilson, S. L. (2020). Intraarticular injection of bone marrow-derived mesenchymal stem cells enhances regeneration in knee osteoarthritis. Knee Surgery, Sports Traumatology, Arthroscopy, 28(12), 3827\u20133842.","journal-title":"Knee Surgery, Sports Traumatology, Arthroscopy"},{"issue":"2","key":"1435_CR66","first-page":"448","volume":"13","author":"C Zhu","year":"2021","unstructured":"Zhu, C., Wu, W., & Qu, X. (2021). Mesenchymal stem cells in osteoarthritis therapy: A review. American Journal of Translational Research, 13(2), 448\u2013461.","journal-title":"American Journal of Translational Research"},{"key":"1435_CR67","doi-asserted-by":"publisher","first-page":"1141601","DOI":"10.3389\/fimmu.2023.1141601","volume":"14","author":"Y Xia","year":"2023","unstructured":"Xia, Y., Zhu, J., Yang, R., Wang, H., Li, Y., & Fu, C. (2023). Mesenchymal stem cells in the treatment of spinal cord injury: Mechanisms, current advances and future challenges. Frontiers in Immunology, 14, 1141601.","journal-title":"Frontiers in Immunology"},{"issue":"1","key":"1435_CR68","doi-asserted-by":"publisher","first-page":"3","DOI":"10.1186\/s13287-024-04127-y","volume":"16","author":"W-C Huang","year":"2025","unstructured":"Huang, W.-C., Li, Y.-C., Chen, P.-X., Ma, K.S.-K., & Wang, L.-T. (2025). Mesenchymal stem cell therapy as a game-changer in liver diseases: Review of current clinical trials. Stem Cell Research and Therapy, 16(1), 3.","journal-title":"Stem Cell Research and Therapy"},{"issue":"5","key":"1435_CR69","doi-asserted-by":"publisher","first-page":"1307","DOI":"10.1634\/stemcells.2006-0561","volume":"25","author":"J-S Bae","year":"2007","unstructured":"Bae, J.-S., Han, H. S., Youn, D.-H., Carter, J. E., Modo, M., Schuchman, E. H., & Jin, H. K. (2007). Bone marrow-derived mesenchymal stem cells promote neuronal networks with functional synaptic transmission after transplantation into mice with neurodegeneration. Stem Cells, 25(5), 1307\u20131316.","journal-title":"Stem Cells"},{"issue":"4","key":"1435_CR70","doi-asserted-by":"publisher","first-page":"821","DOI":"10.1002\/stem.401","volume":"28","author":"H Lee","year":"2010","unstructured":"Lee, H., Lee, J. K., Min, W.-K., Bae, J.-H., He, X., Schuchman, E. H., & Jin, H. K. (2010). Bone marrow-derived mesenchymal stem cells prevent the loss of Niemann\u2013Pick type C mouse Purkinje neurons by correcting sphingolipid metabolism and increasing sphingosine-1-phosphate. Stem Cells, 28(4), 821\u2013831.","journal-title":"Stem Cells"},{"issue":"3","key":"1435_CR71","doi-asserted-by":"publisher","first-page":"234","DOI":"10.1016\/j.neulet.2005.02.029","volume":"381","author":"JS Bae","year":"2005","unstructured":"Bae, J. S., Furuya, S., Ahn, S. J., Yi, S. J., Hirabayashi, Y., & Jin, H. K. (2005). Neuroglial activation in Niemann\u2013Pick Type C mice is suppressed by intracerebral transplantation of bone marrow-derived mesenchymal stem cells. Neuroscience Letters, 381(3), 234\u2013236.","journal-title":"Neuroscience Letters"},{"issue":"9","key":"1435_CR72","doi-asserted-by":"publisher","first-page":"1183","DOI":"10.1172\/JCI14862","volume":"109","author":"HK Jin","year":"2002","unstructured":"Jin, H. K., Carter, J. E., Huntley, G. W., & Schuchman, E. H. (2002). Intracerebral transplantation of mesenchymal stem cells into acid sphingomyelinase-deficient mice delays the onset of neurological abnormalities and extends their life span. Journal of Clinical Investigation, 109(9), 1183\u20131191.","journal-title":"Journal of Clinical Investigation"},{"issue":"6","key":"1435_CR73","doi-asserted-by":"publisher","first-page":"876","DOI":"10.1016\/j.ymthe.2003.07.008","volume":"8","author":"HK Jin","year":"2003","unstructured":"Jin, H. K., & Schuchman, E. H. (2003). Ex vivo gene therapy using bone marrow-derived cells: Combined effects of intracerebral and intravenous transplantation in a mouse model of Niemann\u2013Pick disease. Molecular Therapy, 8(6), 876\u2013885.","journal-title":"Molecular Therapy"},{"key":"1435_CR74","first-page":"1","volume":"2020","author":"J Zhang","year":"2020","unstructured":"Zhang, J., Liu, Y., Chen, Y., Yuan, L., Liu, H., Wang, J., & Li, Y. (2020). Adipose-derived stem cells: Current applications and future directions in the regeneration of multiple tissues. Stem Cells International, 2020, 1\u201326.","journal-title":"Stem Cells International"},{"issue":"2","key":"1435_CR75","doi-asserted-by":"publisher","first-page":"357","DOI":"10.1007\/s00441-010-0942-3","volume":"340","author":"J-S Bae","year":"2010","unstructured":"Bae, J.-S., Carter, J. E., & Jin, H. K. (2010). Adipose tissue-derived stem cells rescue Purkinje neurons and alleviate inflammatory responses in Niemann\u2013Pick disease type C mice. Cell and Tissue Research, 340(2), 357\u2013369.","journal-title":"Cell and Tissue Research"},{"issue":"2","key":"1435_CR76","doi-asserted-by":"publisher","first-page":"887","DOI":"10.1016\/S0014-4886(03)00345-5","volume":"184","author":"M Baudry","year":"2003","unstructured":"Baudry, M., Yao, Y., Simmons, D., Liu, J., & Bi, X. (2003). Postnatal development of inflammation in a murine model of Niemann\u2013Pick type C disease: Immunohistochemical observations of microglia and astroglia. Experimental Neurology, 184(2), 887\u2013903.","journal-title":"Experimental Neurology"},{"issue":"5","key":"1435_CR77","doi-asserted-by":"publisher","first-page":"497","DOI":"10.1177\/0963689718809658","volume":"28","author":"Y Jiao","year":"2018","unstructured":"Jiao, Y., Li, X.-Y., & Liu, J. (2018). A new approach to cerebral palsy treatment: Discussion of the effective components of umbilical cord blood and its mechanisms of action. Cell Transplantation, 28(5), 497\u2013509.","journal-title":"Cell Transplantation"},{"key":"1435_CR78","first-page":"1","volume":"2022","author":"S Gao","year":"2022","unstructured":"Gao, S., Zhang, Y., Liang, K., Bi, R., Du, Y., & Baiocchi, M. (2022). Mesenchymal stem cells (MSCs): A novel therapy for type 2 diabetes. Stem Cells International, 2022, 1\u201317.","journal-title":"Stem Cells International"},{"issue":"7","key":"1435_CR79","doi-asserted-by":"publisher","first-page":"1033","DOI":"10.3727\/096368910X545086","volume":"20","author":"Y Seo","year":"2011","unstructured":"Seo, Y., Yang, S.-R., Jee, M. K., Joo, E. K., Roh, K.-H., Seo, M.-S., & Kang, K.-S. (2011). Human umbilical cord blood-derived mesenchymal stem cells protect against neuronal cell death and ameliorate motor deficits in Niemann Pick Type C1 mice. Cell Transplantation, 20(7), 1033\u20131047.","journal-title":"Cell Transplantation"},{"issue":"6","key":"1435_CR80","doi-asserted-by":"publisher","first-page":"709","DOI":"10.1292\/jvms.09-0495","volume":"72","author":"H Lee","year":"2010","unstructured":"Lee, H., Bae, J. S., & Jin, H. K. (2010). Human umbilical cord blood-derived mesenchymal stem cells improve neurological abnormalities of Niemann\u2013Pick Type C mouse by modulation of neuroinflammatory condition. Journal of Veterinary Medical Science, 72(6), 709\u2013717.","journal-title":"Journal of Veterinary Medical Science"},{"issue":"5","key":"1435_CR81","doi-asserted-by":"publisher","first-page":"630","DOI":"10.3109\/14653249.2012.663485","volume":"14","author":"S-B Hong","year":"2012","unstructured":"Hong, S.-B., Seo, M.-S., Park, S.-B., Seo, Y.-J., Kim, J.-S., & Kang, K.-S. (2012). Therapeutic effects of human amniotic epithelial stem cells in Niemann\u2013Pick type C1 mice. Cytotherapy, 14(5), 630\u2013638.","journal-title":"Cytotherapy"},{"issue":"1","key":"1435_CR82","doi-asserted-by":"publisher","first-page":"406","DOI":"10.1186\/s13287-019-1503-7","volume":"10","author":"L Chen","year":"2019","unstructured":"Chen, L., Qu, J., Cheng, T., Chen, X., & Xiang, C. (2019). Menstrual blood-derived stem cells: Toward therapeutic mechanisms, novel strategies, and future perspectives in the treatment of diseases. Stem Cell Research and Therapy, 10(1), 406.","journal-title":"Stem Cell Research and Therapy"},{"issue":"7","key":"1435_CR83","doi-asserted-by":"publisher","first-page":"2301","DOI":"10.1007\/s11011-023-01243-1","volume":"38","author":"M Yang","year":"2023","unstructured":"Yang, M., Zhao, Y., Li, X., Li, H., Cheng, F., Liu, Y., & Guan, L. (2023). Conditioned medium of human menstrual blood-derived endometrial stem cells protects against cell inflammation and apoptosis of Npc1KO N2a cells. Metabolic Brain Disease, 38(7), 2301\u20132313.","journal-title":"Metabolic Brain Disease"},{"issue":"4","key":"1435_CR84","doi-asserted-by":"publisher","first-page":"791","DOI":"10.3390\/pharmaceutics14040791","volume":"14","author":"D Deo","year":"2022","unstructured":"Deo, D., Marchioni, M., & Rao, P. (2022). Mesenchymal stem\/stromal cells in organ transplantation. Pharmaceutics, 14(4), 791.","journal-title":"Pharmaceutics"},{"issue":"13","key":"1435_CR85","doi-asserted-by":"publisher","first-page":"2925","DOI":"10.3390\/jcm10132925","volume":"10","author":"M Sanchez-Diaz","year":"2021","unstructured":"Sanchez-Diaz, M., Qui\u00f1ones-Vico, M. I., Sanabria de la Torre, R., Montero-V\u00edlchez, T., Sierra-S\u00e1nchez, A., Molina-Leyva, A., & Arias-Santiago, S. (2021). Biodistribution of mesenchymal stromal cells after administration in animal models and humans: A systematic review. Journal of Clinical Medicine, 10(13), 2925.","journal-title":"Journal of Clinical Medicine"},{"issue":"1","key":"1435_CR86","doi-asserted-by":"publisher","first-page":"e77","DOI":"10.1212\/WNL.0000000000007720","volume":"93","author":"W Singer","year":"2019","unstructured":"Singer, W., Dietz, A. B., Zeller, A. D., Gehrking, T. L., Schmelzer, J. D., Schmeichel, A. M., & Low, P. A. (2019). Intrathecal administration of autologous mesenchymal stem cells in multiple system atrophy. Neurology, 93(1), e77\u2013e87.","journal-title":"Neurology"},{"issue":"7","key":"1435_CR87","doi-asserted-by":"publisher","first-page":"2002944","DOI":"10.1002\/advs.202002944","volume":"8","author":"A Andrzejewska","year":"2021","unstructured":"Andrzejewska, A., Dabrowska, S., Lukomska, B., & Janowski, M. (2021). Mesenchymal stem cells for neurological disorders. Advanced Science, 8(7), 2002944.","journal-title":"Advanced Science"},{"issue":"1","key":"1435_CR88","doi-asserted-by":"publisher","first-page":"210","DOI":"10.1186\/s13287-021-02274-0","volume":"12","author":"Y-T Zhang","year":"2021","unstructured":"Zhang, Y.-T., He, K.-J., Zhang, J.-B., Ma, Q.-H., Wang, F., & Liu, C.-F. (2021). Advances in intranasal application of stem cells in the treatment of central nervous system diseases. Stem Cell Research and Therapy, 12(1), 210.","journal-title":"Stem Cell Research and Therapy"},{"key":"1435_CR89","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1155\/2019\/9628536","volume":"2019","author":"B Lukomska","year":"2019","unstructured":"Lukomska, B., Stanaszek, L., Zuba-Surma, E., Legosz, P., Sarzynska, S., & Drela, K. (2019). Challenges and controversies in human mesenchymal stem cell therapy. Stem Cells International, 2019, 1\u201310.","journal-title":"Stem Cells International"},{"key":"1435_CR90","doi-asserted-by":"publisher","first-page":"7","DOI":"10.21037\/sci-2022-025","volume":"9","author":"DS Baranovskii","year":"2022","unstructured":"Baranovskii, D. S., Klabukov, I. D., Arguchinskaya, N. V., Yakimova, A. O., Kisel, A. A., Yatsenko, E. M., & Kaprin, A. D. (2022). Adverse events, side effects and complications in mesenchymal stromal cell-based therapies. Stem Cell Investigation, 9, 7.","journal-title":"Stem Cell Investigation"},{"issue":"1","key":"1435_CR91","doi-asserted-by":"publisher","first-page":"242","DOI":"10.1038\/s41392-024-01936-8","volume":"9","author":"Y Shan","year":"2024","unstructured":"Shan, Y., Zhang, M., Tao, E., Wang, J., Wei, N., Lu, Y., & Wang, G. (2024). Pharmacokinetic characteristics of mesenchymal stem cells in translational challenges. Signal Transduction and Targeted Therapy, 9(1), 242.","journal-title":"Signal Transduction and Targeted Therapy"},{"issue":"3","key":"1435_CR92","doi-asserted-by":"publisher","first-page":"1365","DOI":"10.3390\/ijms25031365","volume":"25","author":"S Gavasso","year":"2024","unstructured":"Gavasso, S., Kr\u00e5kenes, T., Olsen, H., Evjenth, E. C., Ytterdal, M., Haugs\u00f8en, J. B., & Kvistad, C. E. (2024). The therapeutic mechanisms of mesenchymal stem cells in MS\u2014A review focusing on neuroprotective properties. International Journal of Molecular Sciences, 25(3), 1365.","journal-title":"International Journal of Molecular Sciences"},{"issue":"1","key":"1435_CR93","doi-asserted-by":"publisher","first-page":"66","DOI":"10.1186\/s13287-023-03287-7","volume":"14","author":"A Lotfy","year":"2023","unstructured":"Lotfy, A., AboQuella, N. M., & Wang, H. (2023). Mesenchymal stromal\/stem cell (MSC)-derived exosomes in clinical trials. Stem Cell Research and Therapy, 14(1), 66.","journal-title":"Stem Cell Research and Therapy"},{"key":"1435_CR94","doi-asserted-by":"publisher","first-page":"3175","DOI":"10.2147\/IJN.S502591","volume":"20","author":"X Su","year":"2025","unstructured":"Su, X., Wang, H., Li, Q., & Chen, Z. (2025). Extracellular vesicles: A review of their therapeutic potentials, sources, biodistribution, and administration routes. International Journal of Nanomedicine, 20, 3175\u20133199.","journal-title":"International Journal of Nanomedicine"},{"issue":"4","key":"1435_CR95","doi-asserted-by":"publisher","first-page":"836","DOI":"10.1038\/s12276-024-01201-6","volume":"56","author":"HI Kim","year":"2024","unstructured":"Kim, H. I., Park, J., Zhu, Y., Wang, X., Han, Y., & Zhang, D. (2024). Recent advances in extracellular vesicles for therapeutic cargo delivery. Experimental and Molecular Medicine, 56(4), 836\u2013849.","journal-title":"Experimental and Molecular Medicine"},{"issue":"8","key":"1435_CR96","doi-asserted-by":"publisher","first-page":"1959","DOI":"10.3390\/cells10081959","volume":"10","author":"MD Hade","year":"2021","unstructured":"Hade, M. D., Suire, C. N., & Suo, Z. (2021). Mesenchymal stem cell-derived exosomes: Applications in regenerative medicine. Cells, 10(8), 1959.","journal-title":"Cells"},{"key":"1435_CR97","doi-asserted-by":"publisher","first-page":"7923","DOI":"10.2147\/IJN.S444582","volume":"18","author":"J Tian","year":"2023","unstructured":"Tian, J., Han, Z., Song, D., Peng, Y., Xiong, M., Chen, Z., & Zhang, L. (2023). Engineered exosome for drug delivery: Recent development and clinical applications. International Journal of Nanomedicine, 18, 7923\u20137940.","journal-title":"International Journal of Nanomedicine"},{"issue":"2","key":"1435_CR98","doi-asserted-by":"publisher","first-page":"237","DOI":"10.1016\/j.beem.2014.10.002","volume":"29","author":"EH Schuchman","year":"2015","unstructured":"Schuchman, E. H., & Wasserstein, M. P. (2015). Types A and B Niemann\u2013Pick disease. Best Practice and Research: Clinical Endocrinology and Metabolism, 29(2), 237\u2013247.","journal-title":"Best Practice and Research: Clinical Endocrinology and Metabolism"},{"issue":"1","key":"1435_CR99","doi-asserted-by":"publisher","first-page":"140","DOI":"10.1186\/s13023-018-0844-0","volume":"13","author":"M Pineda","year":"2018","unstructured":"Pineda, M., Walterfang, M., & Patterson, M. C. (2018). Miglustat in Niemann\u2013Pick disease type C patients: A review. Orphanet Journal of Rare Diseases, 13(1), 140.","journal-title":"Orphanet Journal of Rare Diseases"},{"issue":"4","key":"1435_CR100","doi-asserted-by":"publisher","first-page":"466","DOI":"10.1016\/j.ymgme.2019.02.004","volume":"126","author":"SE Bianconi","year":"2019","unstructured":"Bianconi, S. E., Hammond, D. I., Farhat, N. Y., Do, A. D., Jenkins, K., Cougnoux, A., & Porter, F. D. (2019). Evaluation of age of death in Niemann\u2013Pick disease, type C: Utility of disease support group websites to understand natural history. Molecular Genetics and Metabolism, 126(4), 466\u2013469.","journal-title":"Molecular Genetics and Metabolism"},{"issue":"4","key":"1435_CR101","doi-asserted-by":"publisher","first-page":"269","DOI":"10.1034\/j.1399-0004.2003.00147.x","volume":"64","author":"MT Vanier","year":"2003","unstructured":"Vanier, M. T., & Millat, G. (2003). Niemann\u2013Pick disease type C. Clinical Genetics, 64(4), 269\u2013281.","journal-title":"Clinical Genetics"},{"issue":"2","key":"1435_CR102","doi-asserted-by":"publisher","first-page":"75","DOI":"10.1177\/088307389801300206","volume":"13","author":"MM Jan","year":"1998","unstructured":"Jan, M. M., & Camfield, P. R. (1998). Nova Scotia Niemann\u2013Pick disease (type D): Clinical study of 20 cases. Journal of Child Neurology, 13(2), 75\u201378.","journal-title":"Journal of Child Neurology"}],"container-title":["Molecular Biotechnology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12033-025-01435-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s12033-025-01435-3","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12033-025-01435-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,30]],"date-time":"2026-03-30T07:02:52Z","timestamp":1774854172000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s12033-025-01435-3"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,4,25]]},"references-count":102,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,3]]}},"alternative-id":["1435"],"URL":"https:\/\/doi.org\/10.1007\/s12033-025-01435-3","relation":{},"ISSN":["1073-6085","1559-0305"],"issn-type":[{"value":"1073-6085","type":"print"},{"value":"1559-0305","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,4,25]]},"assertion":[{"value":"17 July 2024","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"3 April 2025","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"25 April 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}