{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T03:25:45Z","timestamp":1792121145814,"version":"4.4.0"},"reference-count":72,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2021,7,1]],"date-time":"2021-07-01T00:00:00Z","timestamp":1625097600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2021,7,1]],"date-time":"2021-07-01T00:00:00Z","timestamp":1625097600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,6,18]],"date-time":"2021-06-18T00:00:00Z","timestamp":1623974400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["AR055299"],"award-info":[{"award-number":["AR055299"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R01 AR071439"],"award-info":[{"award-number":["R01 AR071439"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008893","name":"University of Iowa","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008893","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100009633","name":"Eunice Kennedy Shriver National Institute of Child Health and Human Development","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100009633","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Cell Reports"],"published-print":{"date-parts":[[2021,7]]},"DOI":"10.1016\/j.celrep.2021.109360","type":"journal-article","created":{"date-parts":[[2021,7,13]],"date-time":"2021-07-13T11:06:24Z","timestamp":1626174384000},"page":"109360","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":23,"title":["A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy"],"prefix":"10.1016","volume":"36","author":[{"given":"Neha R.","family":"Dhoke","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hyunkee","family":"Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sridhar","family":"Selvaraj","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karim","family":"Azzag","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haowen","family":"Zhou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nelio A.J.","family":"Oliveira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sudheer","family":"Tungtur","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolina","family":"Ortiz-Cordero","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Kiley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qi Long","family":"Lu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne G.","family":"Bang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rita C.R.","family":"Perlingeiro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.celrep.2021.109360_bib1","doi-asserted-by":"crossref","first-page":"619","DOI":"10.1016\/j.nmd.2017.02.015","article-title":"Limb girdle muscular dystrophy type 2I: no correlation between clinical severity, histopathology and glycosylated \u03b1-dystroglycan levels in patients homozygous for common FKRP mutation","volume":"27","author":"Alhamidi","year":"2017","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.celrep.2021.109360_bib2","doi-asserted-by":"crossref","first-page":"761","DOI":"10.1016\/j.stemcr.2019.08.014","article-title":"Base-Resolution Methylome of Retinal Pigment Epithelial Cells Used in the First Trial of Human Induced Pluripotent Stem Cell-Based Autologous Transplantation","volume":"13","author":"Araki","year":"2019","journal-title":"Stem Cell Reports"},{"key":"10.1016\/j.celrep.2021.109360_bib3","doi-asserted-by":"crossref","first-page":"1611","DOI":"10.1002\/stem.1402","article-title":"A new immuno-, dystrophin-deficient model, the NSG-mdx(4Cv) mouse, provides evidence for functional improvement following allogeneic satellite cell transplantation","volume":"31","author":"Arpke","year":"2013","journal-title":"Stem Cells"},{"key":"10.1016\/j.celrep.2021.109360_bib4","doi-asserted-by":"crossref","first-page":"2025","DOI":"10.1016\/j.ajpath.2015.03.017","article-title":"Restoration of Functional Glycosylation of \u03b1-Dystroglycan in FKRP Mutant Mice Is Associated with Muscle Regeneration","volume":"185","author":"Awano","year":"2015","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.celrep.2021.109360_bib5","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1186\/s13395-020-00228-3","article-title":"Efficient engraftment of pluripotent stem cell-derived myogenic progenitors in a novel immunodeficient mouse model of limb girdle muscular dystrophy 2I","volume":"10","author":"Azzag","year":"2020","journal-title":"Skelet. Muscle"},{"key":"10.1016\/j.celrep.2021.109360_bib6","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1016\/j.stemcr.2015.08.015","article-title":"cGMP-Manufactured Human Induced Pluripotent Stem Cells Are Available for Pre-clinical and Clinical Applications","volume":"5","author":"Baghbaderani","year":"2015","journal-title":"Stem Cell Reports"},{"key":"10.1016\/j.celrep.2021.109360_bib7","doi-asserted-by":"crossref","first-page":"1033","DOI":"10.1086\/342975","article-title":"Mutations in the O-mannosyltransferase gene POMT1 give rise to the severe neuronal migration disorder Walker-Warburg syndrome","volume":"71","author":"Beltr\u00e1n-Valero de Bernab\u00e9","year":"2002","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib8","doi-asserted-by":"crossref","first-page":"e61","DOI":"10.1136\/jmg.2003.013870","article-title":"Mutations in the FKRP gene can cause muscle-eye-brain disease and Walker-Warburg syndrome","volume":"41","author":"Beltran-Valero de Bernab\u00e9","year":"2004","journal-title":"J.\u00a0Med. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib9","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1083\/jcb.201310035","article-title":"Wnt7a stimulates myogenic stem cell motility and engraftment resulting in improved muscle strength","volume":"205","author":"Bentzinger","year":"2014","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.celrep.2021.109360_bib10","doi-asserted-by":"crossref","first-page":"1047","DOI":"10.1007\/s00428-007-0515-3","article-title":"Biochemical and ultrastructural evidence of endoplasmic reticulum stress in LGMD2I","volume":"451","author":"Boito","year":"2007","journal-title":"Virchows Arch."},{"key":"10.1016\/j.celrep.2021.109360_bib11","doi-asserted-by":"crossref","first-page":"e168","DOI":"10.1093\/nar\/gku936","article-title":"Easy quantitative assessment of genome editing by sequence trace decomposition","volume":"42","author":"Brinkman","year":"2014","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.celrep.2021.109360_bib12","doi-asserted-by":"crossref","first-page":"1198","DOI":"10.1086\/324412","article-title":"Mutations in the fukutin-related protein gene (FKRP) cause a form of congenital muscular dystrophy with secondary laminin \u03b12 deficiency and abnormal glycosylation of \u03b1-dystroglycan","volume":"69","author":"Brockington","year":"2001","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib13","doi-asserted-by":"crossref","first-page":"e14434","DOI":"10.1371\/journal.pone.0014434","article-title":"Transgenic overexpression of LARGE induces \u03b1-dystroglycan hyperglycosylation in skeletal and cardiac muscle","volume":"5","author":"Brockington","year":"2010","journal-title":"PLoS ONE"},{"key":"10.1016\/j.celrep.2021.109360_bib14","doi-asserted-by":"crossref","first-page":"3448","DOI":"10.1038\/s41467-018-05990-z","article-title":"Ribitol restores functionally glycosylated \u03b1-dystroglycan and improves muscle function in dystrophic FKRP-mutant mice","volume":"9","author":"Cataldi","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.celrep.2021.109360_bib15","doi-asserted-by":"crossref","first-page":"3995","DOI":"10.1093\/hmg\/ddq314","article-title":"Fukutin-related protein is essential for mouse muscle, brain and eye development and mutation recapitulates the wide clinical spectrums of dystroglycanopathies","volume":"19","author":"Chan","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib16","doi-asserted-by":"crossref","first-page":"eaaz5618","DOI":"10.1126\/scitranslmed.aaz5618","article-title":"iPSC-derived NK cells maintain high cytotoxicity and enhance in\u00a0vivo tumor control in concert with T\u00a0cells and anti-PD-1 therapy","volume":"12","author":"Cichocki","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.celrep.2021.109360_bib17","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.omtm.2017.11.007","article-title":"Emerging Issues in AAV-Mediated In\u00a0Vivo Gene Therapy","volume":"8","author":"Colella","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.celrep.2021.109360_bib18","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1126\/science.1231143","article-title":"Multiplex genome engineering using CRISPR\/Cas systems","volume":"339","author":"Cong","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.celrep.2021.109360_bib19","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1038\/nm1705","article-title":"Functional skeletal muscle regeneration from differentiating embryonic stem cells","volume":"14","author":"Darabi","year":"2008","journal-title":"Nat. Med."},{"key":"10.1016\/j.celrep.2021.109360_bib20","doi-asserted-by":"crossref","first-page":"610","DOI":"10.1016\/j.stem.2012.02.015","article-title":"Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice","volume":"10","author":"Darabi","year":"2012","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.celrep.2021.109360_bib21","doi-asserted-by":"crossref","first-page":"809","DOI":"10.1083\/jcb.122.4.809","article-title":"A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin","volume":"122","author":"Ervasti","year":"1993","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.celrep.2021.109360_bib22","doi-asserted-by":"crossref","first-page":"519","DOI":"10.1111\/nan.12684","article-title":"Protein homeostasis in LGMDR9 (LGMD2I) \u2013 The role of ubiquitin-proteasome and autophagy-lysosomal system","volume":"47","author":"Franekova","year":"2021","journal-title":"Neuropathol. Appl. Neurobiol."},{"key":"10.1016\/j.celrep.2021.109360_bib23","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1016\/0092-8674(94)90052-3","article-title":"Dystroglycan-alpha, a dystrophin-associated glycoprotein, is a functional agrin receptor","volume":"77","author":"Gee","year":"1994","journal-title":"Cell"},{"key":"10.1016\/j.celrep.2021.109360_bib24","doi-asserted-by":"crossref","first-page":"11534","DOI":"10.1038\/ncomms11534","article-title":"ISPD produces CDP-ribitol used by FKTN and FKRP to transfer ribitol phosphate onto \u03b1-dystroglycan","volume":"7","author":"Gerin","year":"2016","journal-title":"Nat. Commun."},{"key":"10.1016\/j.celrep.2021.109360_bib25","doi-asserted-by":"crossref","first-page":"1952","DOI":"10.1093\/hmg\/ddx066","article-title":"AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression","volume":"26","author":"Gicquel","year":"2017","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib26","doi-asserted-by":"crossref","first-page":"1413","DOI":"10.1093\/glycob\/cws109","article-title":"O-Mannose and O-N-acetyl galactosamine glycosylation of mammalian \u03b1-dystroglycan is conserved in a region-specific manner","volume":"22","author":"Gomez Toledo","year":"2012","journal-title":"Glycobiology"},{"key":"10.1016\/j.celrep.2021.109360_bib27","doi-asserted-by":"crossref","first-page":"2153","DOI":"10.1038\/mt.2012.188","article-title":"Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation","volume":"20","author":"Goudenege","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.celrep.2021.109360_bib28","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1186\/s40659-020-00288-1","article-title":"Engraftment of human induced pluripotent stem cell-derived myogenic progenitors restores dystrophin in mice with duchenne muscular dystrophy","volume":"53","author":"He","year":"2020","journal-title":"Biol. Res."},{"key":"10.1016\/j.celrep.2021.109360_bib29","doi-asserted-by":"crossref","first-page":"1874","DOI":"10.1002\/humu.23827","article-title":"Functional and cellular localization diversity associated with Fukutin-related protein patient genetic variants","volume":"40","author":"Henriques","year":"2019","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.celrep.2021.109360_bib30","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1002\/stem.2230","article-title":"Induced Pluripotent Stem Cell-Derived Natural Killer Cells for Treatment of Ovarian Cancer","volume":"34","author":"Hermanson","year":"2016","journal-title":"Stem Cells"},{"key":"10.1016\/j.celrep.2021.109360_bib31","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1038\/s41556-017-0010-2","article-title":"ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs","volume":"20","author":"Hicks","year":"2018","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.celrep.2021.109360_bib32","article-title":"Inference of CRISPR Edits from Sanger Trace Data","author":"Hsiau","year":"2019","journal-title":"bioRxiv"},{"key":"10.1016\/j.celrep.2021.109360_bib33","doi-asserted-by":"crossref","first-page":"1045","DOI":"10.1038\/s41592-018-0212-y","article-title":"Efficient scarless genome editing in human pluripotent stem cells","volume":"15","author":"Ikeda","year":"2018","journal-title":"Nat. Methods"},{"key":"10.1016\/j.celrep.2021.109360_bib34","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1002\/sctm.20-0242","article-title":"Regulatory considerations for developing a phase I investigational new drug application for autologous induced pluripotent stem cells-based therapy product","volume":"10","author":"Jha","year":"2021","journal-title":"Stem Cells Transl. Med."},{"key":"10.1016\/j.celrep.2021.109360_bib35","doi-asserted-by":"crossref","first-page":"177","DOI":"10.1016\/j.stem.2017.12.010","article-title":"Inhibition of Methyltransferase Setd7 Allows the In\u00a0Vitro Expansion of Myogenic Stem Cells with Improved Therapeutic Potential","volume":"22","author":"Judson","year":"2018","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.celrep.2021.109360_bib36","doi-asserted-by":"crossref","first-page":"2209","DOI":"10.1016\/j.celrep.2016.02.017","article-title":"Identification of a Post-translational Modification with Ribitol-Phosphate and Its Defect in Muscular Dystrophy","volume":"14","author":"Kanagawa","year":"2016","journal-title":"Cell Rep."},{"key":"10.1016\/j.celrep.2021.109360_bib37","doi-asserted-by":"crossref","first-page":"374","DOI":"10.1016\/j.pediatrneurol.2013.06.022","article-title":"Eye and brain abnormalities in congenital muscular dystrophies caused by fukutin-related protein gene (FKRP) mutations","volume":"49","author":"Kava","year":"2013","journal-title":"Pediatr. Neurol."},{"key":"10.1016\/j.celrep.2021.109360_bib38","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.stemcr.2020.11.001","article-title":"Genomic Safe Harbor Expression of PAX7 for the Generation of Engraftable Myogenic Progenitors","volume":"16","author":"Kim","year":"2021","journal-title":"Stem Cell Reports"},{"key":"10.1016\/j.celrep.2021.109360_bib39","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1038\/s41467-019-14220-z","article-title":"Crystal structures of fukutin-related protein (FKRP), a ribitol-phosphate transferase related to muscular dystrophy","volume":"11","author":"Kuwabara","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.celrep.2021.109360_bib40","doi-asserted-by":"crossref","first-page":"e315","DOI":"10.1212\/NXG.0000000000000315","article-title":"Clinical, genetic, and pathologic characterization of FKRP Mexican founder mutation c.1387A>G","volume":"5","author":"Lee","year":"2019","journal-title":"Neurol. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib41","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1038\/s41576-019-0205-4","article-title":"Engineering adeno-associated virus vectors for gene therapy","volume":"21","author":"Li","year":"2020","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib42","doi-asserted-by":"crossref","first-page":"1763","DOI":"10.1093\/hmg\/ddr059","article-title":"Zebrafish Fukutin family proteins link the unfolded protein response with dystroglycanopathies","volume":"20","author":"Lin","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib43","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1007\/s10048-003-0165-9","article-title":"New FKRP mutations causing congenital muscular dystrophy associated with mental retardation and central nervous system abnormalities. Identification of a founder mutation in Tunisian families","volume":"5","author":"Louhichi","year":"2004","journal-title":"Neurogenetics"},{"key":"10.1016\/j.celrep.2021.109360_bib44","doi-asserted-by":"crossref","first-page":"2867","DOI":"10.1016\/j.celrep.2017.06.005","article-title":"PAX7 Targets, CD54, Integrin \u03b19\u03b21, and SDC2, Allow Isolation of Human ESC\/iPSC-Derived Myogenic Progenitors","volume":"19","author":"Magli","year":"2017","journal-title":"Cell Rep."},{"key":"10.1016\/j.celrep.2021.109360_bib45","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1126\/science.1232033","article-title":"RNA-guided human genome engineering via Cas9","volume":"339","author":"Mali","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.celrep.2021.109360_bib46","doi-asserted-by":"crossref","first-page":"417","DOI":"10.2174\/156652407780831601","article-title":"Congenital muscular dystrophies involving the O-mannose pathway","volume":"7","author":"Martin","year":"2007","journal-title":"Curr. Mol. Med."},{"key":"10.1016\/j.celrep.2021.109360_bib47","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1002\/ana.10559","article-title":"Phenotypic spectrum associated with mutations in the fukutin-related protein gene","volume":"53","author":"Mercuri","year":"2003","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.celrep.2021.109360_bib48","doi-asserted-by":"crossref","first-page":"417","DOI":"10.1038\/nature00837","article-title":"Post-translational disruption of dystroglycan-ligand interactions in congenital muscular dystrophies","volume":"418","author":"Michele","year":"2002","journal-title":"Nature"},{"key":"10.1016\/j.celrep.2021.109360_bib49","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1182\/blood-2013-01-306647","article-title":"Immune responses to AAV vectors: overcoming barriers to successful gene therapy","volume":"122","author":"Mingozzi","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.celrep.2021.109360_bib50","doi-asserted-by":"crossref","first-page":"1419","DOI":"10.1016\/S0140-6736(02)11397-3","article-title":"Defective glycosylation in muscular dystrophy","volume":"360","author":"Muntoni","year":"2002","journal-title":"Lancet"},{"key":"10.1016\/j.celrep.2021.109360_bib51","first-page":"129","article-title":"Muscular dystrophies due to defective glycosylation of dystroglycan","volume":"26","author":"Muntoni","year":"2007","journal-title":"Acta Myol."},{"key":"10.1016\/j.celrep.2021.109360_bib52","doi-asserted-by":"crossref","DOI":"10.1242\/dmm.042986","article-title":"Human embryoid bodies as a 3D tissue model of the extracellular matrix and \u03b1-dystroglycanopathies","volume":"13","author":"Nickolls","year":"2020","journal-title":"Dis. Model. Mech."},{"key":"10.1016\/j.celrep.2021.109360_bib53","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1016\/j.tcb.2020.11.003","article-title":"Fukutin-Related Protein: From Pathology to Treatments","volume":"31","author":"Ortiz-Cordero","year":"2021","journal-title":"Trends Cell Biol."},{"key":"10.1016\/j.celrep.2021.109360_bib54","doi-asserted-by":"crossref","first-page":"e65443","DOI":"10.7554\/eLife.65443","article-title":"NAD+ enhances ribitol and ribose rescue of \u03b1-dystroglycan functional glycosylation in human FKRP-mutant myotubes","volume":"10","author":"Ortiz-Cordero","year":"2021","journal-title":"eLife"},{"key":"10.1016\/j.celrep.2021.109360_bib55","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1186\/s13062-015-0035-z","article-title":"\u201cOff-Spotter\u201d: very fast and exhaustive enumeration of genomic lookalikes for designing CRISPR\/Cas guide RNAs","volume":"10","author":"Pliatsika","year":"2015","journal-title":"Biol. Direct"},{"key":"10.1016\/j.celrep.2021.109360_bib56","doi-asserted-by":"crossref","first-page":"1890","DOI":"10.1038\/mt.2014.141","article-title":"Muscle and heart function restoration in a limb girdle muscular dystrophy 2I (LGMD2I) mouse model by systemic FKRP gene delivery","volume":"22","author":"Qiao","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.celrep.2021.109360_bib57","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1038\/s41467-017-02636-4","article-title":"Engineering human pluripotent stem cells into a functional skeletal muscle tissue","volume":"9","author":"Rao","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.celrep.2021.109360_bib58","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1038\/nmeth.2089","article-title":"NIH Image to ImageJ: 25 years of image analysis","volume":"9","author":"Schneider","year":"2012","journal-title":"Nat. Methods"},{"key":"10.1016\/j.celrep.2021.109360_bib59","doi-asserted-by":"crossref","first-page":"777","DOI":"10.1016\/S0092-8674(00)00066-0","article-title":"Pax7 is required for the specification of myogenic satellite cells","volume":"102","author":"Seale","year":"2000","journal-title":"Cell"},{"key":"10.1016\/j.celrep.2021.109360_bib60","doi-asserted-by":"crossref","first-page":"2147","DOI":"10.1016\/j.ymthe.2019.08.011","article-title":"Gene Correction of LGMD2A Patient-Specific iPSCs for the Development of Targeted Autologous Cell Therapy","volume":"27","author":"Selvaraj","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.celrep.2021.109360_bib61","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1016\/j.molmed.2019.07.004","article-title":"Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies","volume":"25","author":"Selvaraj","year":"2019","journal-title":"Trends Mol. Med."},{"key":"10.1016\/j.celrep.2021.109360_bib62","doi-asserted-by":"crossref","first-page":"e47970","DOI":"10.7554\/eLife.47970","article-title":"Screening identifies small molecules that enhance the maturation of human pluripotent stem cell-derived myotubes","volume":"8","author":"Selvaraj","year":"2019","journal-title":"eLife"},{"key":"10.1016\/j.celrep.2021.109360_bib63","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1016\/bs.pbr.2017.02.008","article-title":"Strategies for bringing stem cell-derived dopamine neurons to the clinic--the NYSTEM trial","volume":"230","author":"Studer","year":"2017","journal-title":"Prog. Brain Res."},{"key":"10.1016\/j.celrep.2021.109360_bib64","doi-asserted-by":"crossref","first-page":"140ra89","DOI":"10.1126\/scitranslmed.3003541","article-title":"Transplantation of genetically corrected human iPSC-derived progenitors in mice with limb-girdle muscular dystrophy","volume":"4","author":"Tedesco","year":"2012","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.celrep.2021.109360_bib65","doi-asserted-by":"crossref","first-page":"988","DOI":"10.1212\/01.WNL.0000052996.14099.DC","article-title":"FKRP gene mutations cause congenital muscular dystrophy, mental retardation, and cerebellar cysts","volume":"60","author":"Topaloglu","year":"2003","journal-title":"Neurology"},{"key":"10.1016\/j.celrep.2021.109360_bib66","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1016\/j.omtn.2018.02.008","article-title":"Overexpression of Mutant FKRP Restores Functional Glycosylation and Improves Dystrophic Phenotype in FKRP Mutant Mice","volume":"11","author":"Tucker","year":"2018","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.celrep.2021.109360_bib67","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1089\/hgtb.2013.151","article-title":"Adeno-associated virus-mediated overexpression of LARGE rescues \u03b1-dystroglycan function in dystrophic mice with mutations in the fukutin-related protein","volume":"25","author":"Vannoy","year":"2014","journal-title":"Hum. Gene Ther. Methods"},{"key":"10.1016\/j.celrep.2021.109360_bib68","doi-asserted-by":"crossref","first-page":"1842","DOI":"10.1093\/hmg\/ddt577","article-title":"The transgenic expression of LARGE exacerbates the muscle phenotype of dystroglycanopathy mice","volume":"23","author":"Whitmore","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2021.109360_bib69","doi-asserted-by":"crossref","first-page":"1832","DOI":"10.1038\/mt.2013.156","article-title":"Adeno-associated virus 9 mediated FKRP gene therapy restores functional glycosylation of \u03b1-dystroglycan and improves muscle functions","volume":"21","author":"Xu","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.celrep.2021.109360_bib70","doi-asserted-by":"crossref","first-page":"702","DOI":"10.1093\/glycob\/cwv021","article-title":"Matriglycan: a novel polysaccharide that links dystroglycan to the basement membrane","volume":"25","author":"Yoshida-Moriguchi","year":"2015","journal-title":"Glycobiology"},{"key":"10.1016\/j.celrep.2021.109360_bib71","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1126\/science.1180512","article-title":"O-mannosyl phosphorylation of alpha-dystroglycan is required for laminin binding","volume":"327","author":"Yoshida-Moriguchi","year":"2010","journal-title":"Science"},{"key":"10.1016\/j.celrep.2021.109360_bib72","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1016\/j.stem.2016.01.021","article-title":"A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells","volume":"18","author":"Young","year":"2016","journal-title":"Cell Stem Cell"}],"container-title":["Cell Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2211124721007580?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2211124721007580?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,31]],"date-time":"2025-10-31T11:19:46Z","timestamp":1761909586000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2211124721007580"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,7]]},"references-count":72,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2021,7]]}},"alternative-id":["S2211124721007580"],"URL":"https:\/\/doi.org\/10.1016\/j.celrep.2021.109360","relation":{},"ISSN":["2211-1247"],"issn-type":[{"value":"2211-1247","type":"print"}],"subject":[],"published":{"date-parts":[[2021,7]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy","name":"articletitle","label":"Article Title"},{"value":"Cell Reports","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.celrep.2021.109360","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2021 The Author(s).","name":"copyright","label":"Copyright"}],"article-number":"109360"}}