{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T09:31:59Z","timestamp":1790674319633,"version":"4.1.0"},"reference-count":35,"publisher":"Springer Science and Business Media LLC","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2020,9,22]]},"DOI":"10.7759\/cureus.10600","type":"journal-article","created":{"date-parts":[[2020,9,23]],"date-time":"2020-09-23T03:14:31Z","timestamp":1600830871000},"source":"Crossref","is-referenced-by-count":1,"title":["Duchenne\u2019s Muscular Dystrophy: The Role of Induced Pluripotent Stem Cells and Genomic Editing on Muscle Regeneration"],"prefix":"10.7759","author":[{"given":"Vanessa","family":"May","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashley A","family":"Arnold","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sukrut","family":"Pagad","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manoj R","family":"Somagutta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saijanakan","family":"Sridharan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saruja","family":"Nanthakumaran","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bilal Haider","family":"Malik","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","reference":[{"key":"ref1","doi-asserted-by":"publisher","DOI":"10.3390\/cells7120253","article-title":"Induced pluripotent stem cells for Duchenne muscular dystrophy modeling and therapy","volume":"7","author":"Danisovic L","year":"2018","unstructured":"Danisovic L, Culenova M, Csobonyeiova M. Induced pluripotent stem cells for Duchenne muscular dystrophy modeling and therapy. Cells. 2018, 7:E253. 10.3390\/cells7120253","journal-title":"Cells"},{"key":"ref2","doi-asserted-by":"publisher","DOI":"10.2147\/DNND.S170087","article-title":"Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy - phase I-II","volume":"26","author":"Dai A","year":"2018","unstructured":"Dai A, Baspinar O, Ye\u015filyurt A, et al.. Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy - phase I-II. Degener Neurol Neuromuscul Dis. 2018, 26:63-77. 10.2147\/DNND.S170087","journal-title":"Degener Neurol Neuromuscul Dis"},{"key":"ref3","doi-asserted-by":"publisher","DOI":"10.1097\/PHM.0000000000000138","article-title":"Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine","volume":"94","author":"Braun R","year":"2004","unstructured":"Braun R, Wang Z, Mack D, et al.. Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine. Am J Phys Med Rehabil . 2004, 94:1023-1031. 10.1097\/PHM.0000000000000138","journal-title":"Am J Phys Med Rehabil"},{"key":"ref4","doi-asserted-by":"publisher","DOI":"10.1096\/fj.09-137174","article-title":"Generation of skeletal muscle stem\/progenitor cells from murine induced pluripotent stem cells","volume":"24","author":"Mizuno Y","year":"2010","unstructured":"Mizuno Y, Chang H, Umeda K, et al.. Generation of skeletal muscle stem\/progenitor cells from murine induced pluripotent stem cells. FASEB J. 2010, 24:2245-2253. 10.1096\/fj.09-137174","journal-title":"FASEB J"},{"key":"ref5","doi-asserted-by":"publisher","DOI":"10.1177\/1756286419833478","article-title":"Human induced pluripotent stem cell models for the study and treatment of Duchenne and Becker muscular dystrophies","volume":"12","author":"Piga D","year":"2019","unstructured":"Piga D, Salani S, Magri F, et al.. Human induced pluripotent stem cell models for the study and treatment of Duchenne and Becker muscular dystrophies. Ther Adv Neurol Disord. 2019, 12:1756286419833478. 10.1177\/1756286419833478","journal-title":"Ther Adv Neurol Disord"},{"key":"ref6","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30026-7","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan","volume":"17","author":"Birnkrant DJ","year":"2018","unstructured":"Birnkrant DJ, Bushby K, Bann CM, et al.. Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Lancet Neurol. 2018, 17:445-455. 10.1016\/S1474-4422(18)30026-7","journal-title":"Lancet Neurol"},{"key":"ref7","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000003217","article-title":"Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy","volume":"87","author":"Griggs RC","year":"2016","unstructured":"Griggs RC, Miller JP, Greenberg CR, et al.. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016, 87:2123-2131. 10.1212\/WNL.0000000000003217","journal-title":"Neurology"},{"key":"ref8","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management","volume":"9","author":"Bushby K","year":"2010","unstructured":"Bushby K, Finkel R, Birnkrant DJ, et al.. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010, 9:77-93. 10.1016\/S1474-4422(09)70271-6","journal-title":"Lancet Neurol"},{"key":"ref9","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70272-8","article-title":"Diagnosis and management of Duchenne muscular dystrophy part 2: implementation of multidisciplinary care","volume":"9","author":"Bushby K","year":"2010","unstructured":"Bushby K, Finkel R, Birnkrant DJ, et al.. Diagnosis and management of Duchenne muscular dystrophy part 2: implementation of multidisciplinary care. Lancet Neurol. 2010, 9:177-189. 10.1016\/S1474-4422(09)70272-8","journal-title":"Lancet Neurol"},{"key":"ref10","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2008.05.049","article-title":"Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles","volume":"134","author":"Cerletti M","year":"2008","unstructured":"Cerletti M, Jurga S, Witczak CA, Hirshman MF, Shadrach JL, Goodyear LJ, Wagers AJ. Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles. Cell. 2008, 134:37-47. 10.1016\/j.cell.2008.05.049","journal-title":"Cell"},{"key":"ref11","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.200202092","article-title":"Myogenic specification of side population cells in skeletal muscle","volume":"159","author":"Asakura A","year":"2002","unstructured":"Asakura A, Seale P, Girgis-Gabardo A, Rudnicki MA. Myogenic specification of side population cells in skeletal muscle. J Cell Biol. 2002, 159:123-134. 10.1083\/jcb.200202092","journal-title":"J Cell Biol"},{"key":"ref12","doi-asserted-by":"publisher","DOI":"10.1002\/mus.20560","article-title":"Muscle engraftment of myogenic progenitor cells following intraarterial transplantation","volume":"34","author":"Bachrach E","year":"2006","unstructured":"Bachrach E, Perez AL, Choi YH, et al.. Muscle engraftment of myogenic progenitor cells following intraarterial transplantation. Muscle & Nerve. 2006, 34:44-52. 10.1002\/mus.20560","journal-title":"Muscle & Nerve"},{"key":"ref13","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.125.6.1275","article-title":"Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy","volume":"125","author":"Rando TA","year":"1994","unstructured":"Rando TA, Blau HM. Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J Cell Biol. 1994, 125:1275-1287. 10.1083\/jcb.125.6.1275","journal-title":"J Cell Biol"},{"key":"ref14","doi-asserted-by":"publisher","DOI":"10.1038\/nature07384","article-title":"Self-renewal and expansion of single transplanted muscle stem cells","volume":"456","author":"Sacco A","year":"2008","unstructured":"Sacco A, Doyonnas R, Kraft P, Vitorovic S, Blau HM. Self-renewal and expansion of single transplanted muscle stem cells. Nature. 2008, 456:502-506. 10.1038\/nature07384","journal-title":"Nature"},{"key":"ref15","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.150.5.1085","article-title":"Clonal isolation of muscle-derived cells capable of enhancing muscle regeneration and bone healing","volume":"150","author":"Lee JY","year":"2000","unstructured":"Lee JY, Qu-Peterson Z, Cao B, et al.. Clonal isolation of muscle-derived cells capable of enhancing muscle regeneration and bone healing. J Cell Biol. 2000, 150:1085-1100. 10.1083\/jcb.150.5.1085","journal-title":"J Cell Biol"},{"key":"ref16","doi-asserted-by":"publisher","DOI":"10.1126\/science.1082254","article-title":"Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts","volume":"301","author":"Sampaolesi M","year":"2003","unstructured":"Sampaolesi M, Torrente Y, Innocenzi A, et al.. Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science. 2003, 301:487-492. 10.1126\/science.1082254","journal-title":"Science"},{"key":"ref17","doi-asserted-by":"crossref","DOI":"10.1038\/nature05282","article-title":"Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs","volume":"444","author":"Sampaolesi M","year":"2006","unstructured":"Sampaolesi M, Blot S, D'Antona G, et al.. Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs. Nature. 2006, 444:574-579.","journal-title":"Nature"},{"key":"ref18","doi-asserted-by":"publisher","DOI":"10.1126\/science.1114758","article-title":"Direct isolation of satellite cells for skeletal muscle regeneration","volume":"309","author":"Montarras D","year":"2005","unstructured":"Montarras D, Morgan J, Collins C, et al.. Direct isolation of satellite cells for skeletal muscle regeneration. Science. 2005, 309:2064-2067. 10.1126\/science.1114758","journal-title":"Science"},{"key":"ref19","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2006.07.024","article-title":"Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors","volume":"126","author":"Takahashi K","year":"2006","unstructured":"Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell. 2006, 126:663-676. 10.1016\/j.cell.2006.07.024","journal-title":"Cell"},{"key":"ref20","doi-asserted-by":"publisher","DOI":"10.1016\/j.ncl.2014.05.002","article-title":"Duchenne and Becker muscular dystrophies","volume":"32","author":"Flanigan KM","year":"2014","unstructured":"Flanigan KM. Duchenne and Becker muscular dystrophies. Neurol Clin. 2014, 32:671-688. 10.1016\/j.ncl.2014.05.002","journal-title":"Neurol Clin"},{"key":"ref21","doi-asserted-by":"publisher","DOI":"10.1007\/s00018-013-1396-z","article-title":"Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy","volume":"70","author":"Govoni A","year":"2013","unstructured":"Govoni A, Magri F, Brajkovic S, et al.. Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy. Cell Mol Life Sci. 2013, 70:4585-4602. 10.1007\/s00018-013-1396-z","journal-title":"Cell Mol Life Sci"},{"key":"ref22","doi-asserted-by":"publisher","DOI":"10.1016\/0968-0004(92)90437-E","article-title":"The molecular and biochemical basis of Duchenne muscular dystrophy","volume":"17","author":"Anderson MS","year":"1992","unstructured":"Anderson MS, Kunkel LM. The molecular and biochemical basis of Duchenne muscular dystrophy. Trends Biochem Sci. 1992, 17:289-292. 10.1016\/0968-0004(92)90437-E","journal-title":"Trends Biochem Sci"},{"key":"ref23","doi-asserted-by":"publisher","DOI":"10.1038\/nbt.3297","article-title":"Differentiation of pluripotent stem cells to muscle fiber to model Duchenne muscular dystrophy","volume":"33","author":"Chal J","year":"2015","unstructured":"Chal J, Oginuma M, Al Tanoury Z, et al.. Differentiation of pluripotent stem cells to muscle fiber to model Duchenne muscular dystrophy. Nat Biotechnol. 2015, 33:962-969. 10.1038\/nbt.3297","journal-title":"Nat Biotechnol"},{"key":"ref24","doi-asserted-by":"publisher","DOI":"10.4172\/2157-7633.1000361","article-title":"Induced pluripotent stem cells derived muscle progenitors effectively mitigate muscular dystrophy through restoring the dystrophin distribution","volume":"6","author":"Cai","year":"2016","unstructured":"Cai, Huang, Wang, et al.. Induced pluripotent stem cells derived muscle progenitors effectively mitigate muscular dystrophy through restoring the dystrophin distribution. J Stem Cell Res Ther. 2016, 6:1000361. 10.4172\/2157-7633.1000361","journal-title":"J Stem Cell Res Ther"},{"key":"ref25","doi-asserted-by":"publisher","DOI":"10.1007\/s40778-017-0076-6","article-title":"Genome editing and muscle stem cells as a therapeutic tool for muscular dystrophies","volume":"3","author":"Pini","year":"2017","unstructured":"Pini, Morgan, Muntoni, et al.. Genome editing and muscle stem cells as a therapeutic tool for muscular dystrophies. J Stem Cell Res Ther. 2017, 3:137-148. 10.1007\/s40778-017-0076-6","journal-title":"J Stem Cell Res Ther"},{"key":"ref26","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbadis.2014.07.023","article-title":"Spectrum of muscular dystrophies associated with sarcolemmal-protein genetic defects","volume":"1852","author":"Nigro V","year":"2015","unstructured":"Nigro V, Piluso G. Spectrum of muscular dystrophies associated with sarcolemmal-protein genetic defects. Biochem Biophys Acta. 2015, 1852:585-593. 10.1016\/j.bbadis.2014.07.023","journal-title":"Biochem Biophys Acta"},{"key":"ref27","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e31825f04ea","article-title":"Importance of SPP1 genotype as a covariate in clinical trials in Duchenne muscular dystrophy","volume":"79","author":"Bello L","year":"2012","unstructured":"Bello L, Piva L, Barp A, et al.. Importance of SPP1 genotype as a covariate in clinical trials in Duchenne muscular dystrophy. Neurology. 2012, 79:159-162. 10.1212\/WNL.0b013e31825f04ea","journal-title":"Neurology"},{"key":"ref28","doi-asserted-by":"publisher","DOI":"10.1007\/s12015-017-9792-7","article-title":"Creation of dystrophin expressing chimeric cells of myoblast origin as a novel stem cell based therapy for Duchenne muscular dystrophy","volume":"14","author":"Siemionow","year":"2018","unstructured":"Siemionow, Cwykiel, Heydemann, et al.. Creation of dystrophin expressing chimeric cells of myoblast origin as a novel stem cell based therapy for Duchenne muscular dystrophy. Stem Cell Rev and Rep. 2018, 14:189-199. 10.1007\/s12015-017-9792-7","journal-title":"Stem Cell Rev and Rep"},{"key":"ref29","doi-asserted-by":"crossref","DOI":"10.1038\/srep12831","article-title":"Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells","volume":"5","author":"Shoji E","year":"2015","unstructured":"Shoji E, Sakurai H, Nishino T, et al.. Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells. Sci Rep. 2015, 5:12831.","journal-title":"Sci Rep"},{"key":"ref30","doi-asserted-by":"publisher","DOI":"10.1126\/science.aad5177","article-title":"In vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"Tabebordbar M","year":"2016","unstructured":"Tabebordbar M, Zhu K, Cheng JKW, et al.. In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science. 2016, 351:407-411. 10.1126\/science.aad5177","journal-title":"Science"},{"key":"ref31","doi-asserted-by":"publisher","DOI":"10.1126\/sciadv.aav4324","article-title":"CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells","volume":"5","author":"Min YL","year":"2019","unstructured":"Min YL, Li H, Rodriguez-Caycedo C et al.. CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells. Sci Adv. 2019, 5:eaav4324. 10.1126\/sciadv.aav4324","journal-title":"Sci Adv"},{"key":"ref32","doi-asserted-by":"publisher","DOI":"10.1016\/j.celrep.2016.05.016","article-title":"Concordant but varied phenotypes among duchenne muscular dystrophy patient-specific myoblasts derived using a human iPSC-based model","volume":"15","author":"Choi IY","year":"2016","unstructured":"Choi IY, Lim H, Estrellas K, et al.. Concordant but varied phenotypes among duchenne muscular dystrophy patient-specific myoblasts derived using a human iPSC-based model. Cell Rep. 2016, 15:2301-2312. 10.1016\/j.celrep.2016.05.016","journal-title":"Cell Rep"},{"key":"ref33","doi-asserted-by":"publisher","DOI":"10.1007\/s12015-011-9258-2","article-title":"Functional myogenic engraftment from mouse iPS cells","volume":"7","author":"Radbod Darabi","year":"2011","unstructured":"Radbod Darabi, Weihong Pan, Darko Bosnakovski, et al.. Functional myogenic engraftment from mouse iPS cells. Stem Cell Rev Rep. 2011, 7:948-957. 10.1007\/s12015-011-9258-2","journal-title":"Stem Cell Rev Rep"},{"key":"ref34","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2008.05.049","article-title":"Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles","volume":"134","author":"Cerletti M","year":"2008","unstructured":"Cerletti M, Jurga S, Witczak CA et al.. Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles. Cell. 2008, 134:37-47. 10.1016\/j.cell.2008.05.049","journal-title":"Cell"},{"key":"ref35","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2017.10.013","article-title":"Skeletal muscle regenerative potential of human MuStem cells following transplantation\r\ninto injured mice muscle","volume":"7","author":"Lorant J","year":"2018","unstructured":"Lorant J, Saury C, Schleder C, et al.. Skeletal muscle regenerative potential of human MuStem cells following transplantation\r\ninto injured mice muscle. Mol Ther. 2018, 7:8. 10.1016\/j.ymthe.2017.10.013","journal-title":"Mol Ther"}],"container-title":["Cureus"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.cureus.com\/articles\/37366-duchennes-muscular-dystrophy-the-role-of-induced-pluripotent-stem-cells-and-genomic-editing-on-muscle-regeneration","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,2,9]],"date-time":"2024-02-09T19:26:34Z","timestamp":1707506794000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.cureus.com\/articles\/37366-duchennes-muscular-dystrophy-the-role-of-induced-pluripotent-stem-cells-and-genomic-editing-on-muscle-regeneration"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,9,22]]},"references-count":35,"URL":"https:\/\/doi.org\/10.7759\/cureus.10600","relation":{},"ISSN":["2168-8184"],"issn-type":[{"value":"2168-8184","type":"print"}],"subject":[],"published":{"date-parts":[[2020,9,22]]}}}