{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T21:21:31Z","timestamp":1783027291851,"version":"3.54.6"},"reference-count":63,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,4,16]],"date-time":"2026-04-16T00:00:00Z","timestamp":1776297600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001279","name":"Great Ormond Street Hospital Charity","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001279","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000272","name":"National Institute for Health and Care Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000272","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003784","name":"Great Ormond Street Hospital for Children","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003784","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000765","name":"University College London","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000765","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002108","name":"Friedreich's Ataxia Research Alliance","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010269","name":"Wellcome Trust","doi-asserted-by":"publisher","award":["217112\/Z\/19\/Z"],"award-info":[{"award-number":["217112\/Z\/19\/Z"]}],"id":[{"id":"10.13039\/100010269","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Cell Reports Medicine"],"published-print":{"date-parts":[[2026,6]]},"DOI":"10.1016\/j.xcrm.2026.102803","type":"journal-article","created":{"date-parts":[[2026,5,14]],"date-time":"2026-05-14T13:52:34Z","timestamp":1778766754000},"page":"102803","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich\u2019s ataxia models"],"prefix":"10.1016","volume":"7","author":[{"given":"Jeffrey","family":"Pido-Lopez","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shefta E","family":"Moula","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Enas","family":"Shaban","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Konstantinos","family":"Stamatiou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bethan J.","family":"Critchley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas E.","family":"Whittaker","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stina","family":"Svensson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Anjomani-Virmouni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ester","family":"Kalef-Ezra","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucinda","family":"Carr","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jane","family":"Hassel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian J.","family":"Thrasher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manju A.","family":"Kurian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ian A.","family":"Blair","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Teerapat","family":"Rojsajjakul","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giorgia","family":"Santilli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2841-7866","authenticated-orcid":false,"given":"Arturo","family":"Sala","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.xcrm.2026.102803_bib1","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1111\/jnc.12317","article-title":"Clinical features of Friedreich's ataxia: classical and atypical phenotypes","volume":"126","author":"Parkinson","year":"2013","journal-title":"J. Neurochem."},{"key":"10.1016\/j.xcrm.2026.102803_bib2","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1086\/301680","article-title":"The GAA triplet-repeat expansion in Friedreich ataxia interferes with transcription and may be associated with an unusual DNA structure","volume":"62","author":"Bidichandani","year":"1998","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.xcrm.2026.102803_bib3","doi-asserted-by":"crossref","first-page":"1423","DOI":"10.1126\/science.271.5254.1423","article-title":"Friedreich\u2019s ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion","volume":"271","author":"Campuzano","year":"1996","journal-title":"Science"},{"key":"10.1016\/j.xcrm.2026.102803_bib4","doi-asserted-by":"crossref","first-page":"7767","DOI":"10.1021\/bi060424r","article-title":"Monomeric yeast frataxin is an iron-binding protein","volume":"45","author":"Cook","year":"2006","journal-title":"Biochemistry"},{"key":"10.1016\/j.xcrm.2026.102803_bib5","doi-asserted-by":"crossref","first-page":"467","DOI":"10.1093\/hmg\/ddi461","article-title":"Mitochondrial iron detoxification is a primary function of frataxin that limits oxidative damage and preserves cell longevity","volume":"15","author":"Gakh","year":"2006","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.xcrm.2026.102803_bib6","doi-asserted-by":"crossref","first-page":"779","DOI":"10.1002\/ana.25207","article-title":"Bone marrow transplantation stimulates neural repair in Friedreich's ataxia mice","volume":"83","author":"Kemp","year":"2018","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.xcrm.2026.102803_bib7","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aaj2347","article-title":"Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich's ataxia","volume":"9","author":"Rocca","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.xcrm.2026.102803_bib8","doi-asserted-by":"crossref","first-page":"667","DOI":"10.1056\/NEJM199103073241005","article-title":"Graft-versus-host disease","volume":"324","author":"Ferrara","year":"1991","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.xcrm.2026.102803_bib9","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1007\/s00467-008-1020-x","article-title":"Mechanism of cellular rejection in transplantation","volume":"25","author":"Ingulli","year":"2010","journal-title":"Pediatr. Nephrol."},{"key":"10.1016\/j.xcrm.2026.102803_bib10","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1084\/jem.158.1.1","article-title":"Graft-versus-host disease in cyclosporin A-treated rats after syngeneic and autologous bone marrow reconstitution","volume":"158","author":"Glazier","year":"1983","journal-title":"J. Exp. Med."},{"key":"10.1016\/j.xcrm.2026.102803_bib11","doi-asserted-by":"crossref","first-page":"409","DOI":"10.1038\/bmt.2009.157","article-title":"A case of severe cutaneous, GI and liver GVHD in a patient with multiple myeloma, status-post-second auto-SCT","volume":"45","author":"Goddard","year":"2010","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.xcrm.2026.102803_bib12","doi-asserted-by":"crossref","DOI":"10.1016\/j.jim.2019.07.001","article-title":"Evaluation of antibodies for western blot analysis of frataxin protein isoforms","volume":"474","author":"Weng","year":"2019","journal-title":"J. Immunol. Methods"},{"key":"10.1016\/j.xcrm.2026.102803_bib13","doi-asserted-by":"crossref","first-page":"3521","DOI":"10.1093\/hmg\/ddn244","article-title":"The in vivo mitochondrial two-step maturation of human frataxin","volume":"17","author":"Schmucker","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.xcrm.2026.102803_bib14","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1007\/s00232-016-9940-z","article-title":"Progress in Research and Application of HIV-1 TAT-Derived Cell-Penetrating Peptide","volume":"250","author":"Zou","year":"2017","journal-title":"J. Membr. Biol."},{"key":"10.1016\/j.xcrm.2026.102803_bib15","doi-asserted-by":"crossref","DOI":"10.1038\/srep11719","article-title":"Discovery of a non-cationic cell penetrating peptide derived from membrane-interacting human proteins and its potential as a protein delivery carrier","volume":"5","author":"Young Kim","year":"2015","journal-title":"Sci. Rep."},{"key":"10.1016\/j.xcrm.2026.102803_bib16","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1093\/hmg\/8.3.425","article-title":"The Friedreich's ataxia mutation confers cellular sensitivity to oxidant stress which is rescued by chelators of iron and calcium and inhibitors of apoptosis","volume":"8","author":"Wong","year":"1999","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.xcrm.2026.102803_bib17","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1093\/hmg\/ddn308","article-title":"Mitochondrial ferritin limits oxidative damage regulating mitochondrial iron availability: hypothesis for a protective role in Friedreich ataxia","volume":"18","author":"Campanella","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.xcrm.2026.102803_bib18","doi-asserted-by":"crossref","first-page":"4138","DOI":"10.1073\/pnas.082108799","article-title":"From bacteria to mitochondria: aconitase yields surprises","volume":"99","author":"Walden","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.xcrm.2026.102803_bib19","first-page":"608","article-title":"Neurons and cardiomyocytes derived from induced pluripotent stem cells as a model for mitochondrial defects in Friedreich's ataxia","volume":"6","author":"Hick","year":"2013","journal-title":"Dis. Model. Mech."},{"key":"10.1016\/j.xcrm.2026.102803_bib20","doi-asserted-by":"crossref","first-page":"1594","DOI":"10.1093\/hmg\/ddy448","article-title":"GRP75 overexpression rescues frataxin deficiency and mitochondrial phenotypes in Friedreich ataxia cellular models","volume":"28","author":"Dong","year":"2019","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.xcrm.2026.102803_bib21","doi-asserted-by":"crossref","first-page":"75","DOI":"10.3389\/fnins.2019.00075","article-title":"The Role of Iron in Friedreich's Ataxia: Insights From Studies in Human Tissues and Cellular and Animal Models","volume":"13","author":"Llorens","year":"2019","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.xcrm.2026.102803_bib22","doi-asserted-by":"crossref","first-page":"9757","DOI":"10.1073\/pnas.0804261105","article-title":"The MCK mouse heart model of Friedreich's ataxia: Alterations in iron-regulated proteins and cardiac hypertrophy are limited by iron chelation","volume":"105","author":"Whitnall","year":"2008","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.xcrm.2026.102803_bib23","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1016\/j.mcn.2012.08.003","article-title":"Iron-sulfur cluster synthesis, iron homeostasis and oxidative stress in Friedreich ataxia","volume":"55","author":"Vaubel","year":"2013","journal-title":"Mol. Cell. Neurosci."},{"key":"10.1016\/j.xcrm.2026.102803_bib24","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1016\/S0140-6736(21)02017-1","article-title":"Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1\/2 trial and expanded access","volume":"399","author":"Fumagalli","year":"2022","journal-title":"Lancet"},{"key":"10.1016\/j.xcrm.2026.102803_bib25","doi-asserted-by":"crossref","first-page":"200","DOI":"10.1038\/s41591-019-0735-5","article-title":"Lentiviral gene therapy for X-linked chronic granulomatous disease","volume":"26","author":"Kohn","year":"2020","journal-title":"Nat. Med."},{"key":"10.1016\/j.xcrm.2026.102803_bib26","doi-asserted-by":"crossref","first-page":"2002","DOI":"10.1056\/NEJMoa2027675","article-title":"Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency","volume":"384","author":"Kohn","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.xcrm.2026.102803_bib27","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1038\/s41591-021-01641-x","article-title":"Long-term safety and efficacy of lentiviral hematopoietic stem\/progenitor cell gene therapy for Wiskott-Aldrich syndrome","volume":"28","author":"Magnani","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.xcrm.2026.102803_bib28","doi-asserted-by":"crossref","first-page":"949","DOI":"10.1089\/hum.2020.276","article-title":"Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47(phox)-Deficient Chronic Granulomatous Disease","volume":"32","author":"Schejtman","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.xcrm.2026.102803_bib29","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0089488","article-title":"Generation and characterisation of Friedreich ataxia YG8R mouse fibroblast and neural stem cell models","volume":"9","author":"Sandi","year":"2014","journal-title":"PLoS One"},{"key":"10.1016\/j.xcrm.2026.102803_bib30","first-page":"225","article-title":"A novel GAA-repeat-expansion-based mouse model of Friedreich's ataxia","volume":"8","author":"Anjomani","year":"2015","journal-title":"Dis. Model. Mech."},{"key":"10.1016\/j.xcrm.2026.102803_bib31","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2022.819569","article-title":"Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia","volume":"16","author":"Mercado-Ay\u00f3n","year":"2022","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.xcrm.2026.102803_bib32","doi-asserted-by":"crossref","first-page":"323","DOI":"10.1007\/s00401-011-0844-9","article-title":"The cerebellar component of Friedreich's ataxia","volume":"122","author":"Koeppen","year":"2011","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.xcrm.2026.102803_bib33","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1038\/ng1097-215","article-title":"Aconitase and mitochondrial iron-sulphur protein deficiency in Friedreich ataxia","volume":"17","author":"R\u00f6tig","year":"1997","journal-title":"Nat. Genet."},{"key":"10.1016\/j.xcrm.2026.102803_bib34","doi-asserted-by":"crossref","first-page":"580","DOI":"10.1016\/j.ygeno.2006.06.015","article-title":"GAA repeat expansion mutation mouse models of Friedreich ataxia exhibit oxidative stress leading to progressive neuronal and cardiac pathology","volume":"88","author":"Al-Mahdawi","year":"2006","journal-title":"Genomics"},{"key":"10.1016\/j.xcrm.2026.102803_bib35","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1111\/j.1469-8749.2011.03931.x","article-title":"In children with Friedreich ataxia, muscle and ataxia parameters are associated","volume":"53","author":"Sival","year":"2011","journal-title":"Dev. Med. Child Neurol."},{"key":"10.1016\/j.xcrm.2026.102803_bib36","doi-asserted-by":"crossref","first-page":"708","DOI":"10.1097\/NEN.0b013e31825fed76","article-title":"Skeletal muscle involvement in friedreich ataxia and potential effects of recombinant human erythropoietin administration on muscle regeneration and neovascularization","volume":"71","author":"Nachbauer","year":"2012","journal-title":"J. Neuropathol. Exp. Neurol."},{"key":"10.1016\/j.xcrm.2026.102803_bib37","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1016\/j.ijcard.2021.11.033","article-title":"Cellular pathophysiology of Friedreich's ataxia cardiomyopathy","volume":"346","author":"Lees","year":"2022","journal-title":"Int. J. Cardiol."},{"key":"10.1016\/j.xcrm.2026.102803_bib38","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1016\/j.omtm.2020.08.018","article-title":"High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models","volume":"19","author":"Belbellaa","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.xcrm.2026.102803_bib39","doi-asserted-by":"crossref","first-page":"935","DOI":"10.1002\/mds.25836","article-title":"Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia","volume":"29","author":"Boesch","year":"2014","journal-title":"Mov. Disord."},{"key":"10.1016\/j.xcrm.2026.102803_bib40","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1093\/hmg\/ddq566","article-title":"Preventing the ubiquitin-proteasome-dependent degradation of frataxin, the protein defective in Friedreich's ataxia","volume":"20","author":"Rufini","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.xcrm.2026.102803_bib41","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1007\/s12311-008-0084-2","article-title":"Friedreich's Ataxia: from the (GAA)n repeat mediated silencing to new promising molecules for therapy","volume":"8","author":"Marmolino","year":"2009","journal-title":"Cerebellum"},{"key":"10.1016\/j.xcrm.2026.102803_bib42","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1111\/jnc.12300","article-title":"Deferiprone for the treatment of Friedreich's ataxia","volume":"126","author":"Pandolfo","year":"2013","journal-title":"J. Neurochem."},{"key":"10.1016\/j.xcrm.2026.102803_bib43","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1007\/s00702-004-0216-x","article-title":"L-carnitine and creatine in Friedreich's ataxia. A randomized, placebo-controlled crossover trial","volume":"112","author":"Sch\u00f6ls","year":"2005","journal-title":"J. Neural Transm."},{"key":"10.1016\/j.xcrm.2026.102803_bib44","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1001\/archneurol.2010.168","article-title":"A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia","volume":"67","author":"Lynch","year":"2010","journal-title":"Arch. Neurol."},{"key":"10.1016\/j.xcrm.2026.102803_bib45","doi-asserted-by":"crossref","first-page":"590","DOI":"10.1002\/ana.1001","article-title":"Antioxidant treatment improves in vivo cardiac and skeletal muscle bioenergetics in patients with Friedreich\u2019s ataxia","volume":"49","author":"Lodi","year":"2001","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.xcrm.2026.102803_bib46","doi-asserted-by":"crossref","first-page":"3106","DOI":"10.1016\/j.neuron.2024.06.029","article-title":"Microglia rescue neurons from aggregate-induced neuronal dysfunction and death through tunneling nanotubes","volume":"112","author":"Scheiblich","year":"2024","journal-title":"Neuron"},{"key":"10.1016\/j.xcrm.2026.102803_bib47","doi-asserted-by":"crossref","first-page":"15018","DOI":"10.1073\/pnas.1205858109","article-title":"Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation","volume":"109","author":"Capotondo","year":"2012","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.xcrm.2026.102803_bib48","doi-asserted-by":"crossref","first-page":"18150","DOI":"10.1073\/pnas.1210150109","article-title":"Microglial repopulation model reveals a robust homeostatic process for replacing CNS myeloid cells","volume":"109","author":"Varvel","year":"2012","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.xcrm.2026.102803_bib49","doi-asserted-by":"crossref","first-page":"4845","DOI":"10.1038\/s41467-018-07295-7","article-title":"Competitive repopulation of an empty microglial niche yields functionally distinct subsets of microglia-like cells","volume":"9","author":"Lund","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.xcrm.2026.102803_bib50","doi-asserted-by":"crossref","first-page":"33","DOI":"10.12968\/hmed.2019.80.1.33","article-title":"Haematopoietic stem cell transplants: principles and indications","volume":"80","author":"Balassa","year":"2019","journal-title":"Br. J. Hosp. Med."},{"key":"10.1016\/j.xcrm.2026.102803_bib51","doi-asserted-by":"crossref","first-page":"745","DOI":"10.1001\/archderm.1987.01660300067014","article-title":"Acute graft-vs-host disease. Development following autologous and syngeneic bone marrow transplantation","volume":"123","author":"Hood","year":"1987","journal-title":"Arch. Dermatol."},{"key":"10.1016\/j.xcrm.2026.102803_bib52","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1038\/bmt.2009.255","article-title":"Background to hematopoietic cell transplantation, including post transplant immune recovery","volume":"44","author":"Mackall","year":"2009","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.xcrm.2026.102803_bib53","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1016\/j.omtm.2022.02.002","article-title":"In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency","volume":"24","author":"Huichalaf","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.xcrm.2026.102803_bib54","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1111\/jcmm.13365","article-title":"Frataxin-deficient neurons and mice models of Friedreich ataxia are improved by TAT-MTScs-FXN treatment","volume":"22","author":"Britti","year":"2018","journal-title":"J. Cell Mol. Med."},{"key":"10.1016\/j.xcrm.2026.102803_bib55","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1517\/14712598.8.4.397","article-title":"Gene therapy of inherited immunodeficiencies","volume":"8","author":"Santilli","year":"2008","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.xcrm.2026.102803_bib56","doi-asserted-by":"crossref","DOI":"10.1126\/science.1233151","article-title":"Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome","volume":"341","author":"Aiuti","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.xcrm.2026.102803_bib57","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0107416","article-title":"Cellular, molecular and functional characterisation of YAC transgenic mouse models of Friedreich ataxia","volume":"9","author":"Anjomani Virmouni","year":"2014","journal-title":"PLoS One"},{"key":"10.1016\/j.xcrm.2026.102803_bib58","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2023.930422","article-title":"A new FRDA mouse model [Fxn (null):YG8s(GAA) > 800] with more than 800 GAA repeats","volume":"17","author":"Kalef-Ezra","year":"2023","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.xcrm.2026.102803_bib59","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-018-29792-x","article-title":"In vivo neutralization of the protagonist role of macrophages during the chronic inflammatory stage of Huntington's disease","volume":"8","author":"Pido-Lopez","year":"2018","journal-title":"Sci. Rep."},{"key":"10.1016\/j.xcrm.2026.102803_bib60","doi-asserted-by":"crossref","first-page":"7202","DOI":"10.1038\/s41598-019-43627-3","article-title":"Inhibition of tumour necrosis factor alpha in the R6\/2 mouse model of Huntington's disease by etanercept treatment","volume":"9","author":"Pido-Lopez","year":"2019","journal-title":"Sci. Rep."},{"key":"10.1016\/j.xcrm.2026.102803_bib61","doi-asserted-by":"crossref","first-page":"8391","DOI":"10.1038\/s41598-024-59060-0","article-title":"Expression and processing of mature human frataxin after gene therapy in mice","volume":"14","author":"Rojsajjakul","year":"2024","journal-title":"Sci. Rep."},{"key":"10.1016\/j.xcrm.2026.102803_bib62","doi-asserted-by":"crossref","first-page":"4251","DOI":"10.1021\/acs.analchem.3c00091","article-title":"Liquid Chromatography-Mass Spectrometry Analysis of Frataxin Proteoforms in Whole Blood as Biomarkers of the Genetic Disease Friedreich's Ataxia","volume":"95","author":"Rojsajjakul","year":"2023","journal-title":"Anal. Chem."},{"key":"10.1016\/j.xcrm.2026.102803_bib63","doi-asserted-by":"crossref","first-page":"966","DOI":"10.1093\/bioinformatics\/btq054","article-title":"Skyline: an open source document editor for creating and analyzing targeted proteomics experiments","volume":"26","author":"MacLean","year":"2010","journal-title":"Bioinformatics"}],"container-title":["Cell Reports Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S266637912600220X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S266637912600220X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T20:53:30Z","timestamp":1783025610000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S266637912600220X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,6]]},"references-count":63,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2026,6]]}},"alternative-id":["S266637912600220X"],"URL":"https:\/\/doi.org\/10.1016\/j.xcrm.2026.102803","relation":{},"ISSN":["2666-3791"],"issn-type":[{"value":"2666-3791","type":"print"}],"subject":[],"published":{"date-parts":[[2026,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich\u2019s ataxia models","name":"articletitle","label":"Article Title"},{"value":"Cell Reports Medicine","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.xcrm.2026.102803","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Author(s). Published by Elsevier Inc.","name":"copyright","label":"Copyright"}],"article-number":"102803"}}