{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,18]],"date-time":"2026-03-18T05:27:28Z","timestamp":1773811648813,"version":"3.50.1"},"reference-count":42,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2022,9,30]],"date-time":"2022-09-30T00:00:00Z","timestamp":1664496000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2022,12]]},"DOI":"10.1016\/j.omtm.2022.09.014","type":"journal-article","created":{"date-parts":[[2022,10,3]],"date-time":"2022-10-03T12:17:41Z","timestamp":1664799461000},"page":"206-216","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":25,"special_numbering":"C","title":["Gene therapy restores adipose tissue and metabolic health in a pre-clinical mouse model of lipodystrophy"],"prefix":"10.1016","volume":"27","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2380-7117","authenticated-orcid":false,"given":"Nadine","family":"Sommer","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6831-4426","authenticated-orcid":false,"given":"Ahlima","family":"Roumane","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Weiping","family":"Han","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6193-3152","authenticated-orcid":false,"given":"Mirela","family":"Delibegovi\u0107","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5020-4939","authenticated-orcid":false,"given":"Justin J.","family":"Rochford","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4230-4355","authenticated-orcid":false,"given":"George D.","family":"Mcilroy","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2022.09.014_bib1","doi-asserted-by":"crossref","first-page":"eaan4672","DOI":"10.1126\/science.aan4672","article-title":"Gene therapy comes of age","volume":"359","author":"Dunbar","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.omtm.2022.09.014_bib2","doi-asserted-by":"crossref","first-page":"20","DOI":"10.1016\/j.cell.2013.12.012","article-title":"What we talk about when we talk about fat","volume":"156","author":"Rosen","year":"2014","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2022.09.014_bib3","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1016\/j.pathol.2018.11.004","article-title":"Lipodystrophies, dyslipidaemias and atherosclerotic cardiovascular disease","volume":"51","author":"Hussain","year":"2019","journal-title":"Pathology"},{"key":"10.1016\/j.omtm.2022.09.014_bib4","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1038\/nrendo.2015.123","article-title":"Congenital generalized lipodystrophies--new insights into metabolic dysfunction","volume":"11","author":"Patni","year":"2015","journal-title":"Nat. Rev. Endocrinol."},{"key":"10.1016\/j.omtm.2022.09.014_bib5","doi-asserted-by":"crossref","first-page":"2759","DOI":"10.1210\/jc.2016-1005","article-title":"Natural history of congenital generalized lipodystrophy: a nationwide study from Turkey","volume":"101","author":"Akinci","year":"2016","journal-title":"J.\u00a0Clin. Endocrinol. Metab."},{"key":"10.1016\/j.omtm.2022.09.014_bib6","doi-asserted-by":"crossref","first-page":"375","DOI":"10.2147\/DMSO.S130810","article-title":"Estimating the prevalence of generalized and partial lipodystrophy: findings and challenges","volume":"10","author":"Chiquette","year":"2017","journal-title":"Diabetes Metab. Syndr. Obes."},{"key":"10.1016\/j.omtm.2022.09.014_bib7","doi-asserted-by":"crossref","first-page":"365","DOI":"10.1038\/ng585","article-title":"Identification of the gene altered in Berardinelli-Seip congenital lipodystrophy on chromosome 11q13","volume":"28","author":"Magr\u00e9","year":"2001","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtm.2022.09.014_bib8","doi-asserted-by":"crossref","first-page":"3022","DOI":"10.1093\/hmg\/ddr205","article-title":"Seipin ablation in mice results in severe generalized lipodystrophy","volume":"20","author":"Cui","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2022.09.014_bib9","doi-asserted-by":"crossref","first-page":"1099","DOI":"10.1128\/MCB.06465-11","article-title":"Berardinelli-seip congenital lipodystrophy 2\/seipin is a cell-autonomous regulator of lipolysis essential for adipocyte differentiation","volume":"32","author":"Chen","year":"2012","journal-title":"Mol. Cell Biol."},{"key":"10.1016\/j.omtm.2022.09.014_bib10","doi-asserted-by":"crossref","first-page":"1813","DOI":"10.1007\/s00125-013-2926-9","article-title":"Thiazolidinediones partially reverse the metabolic disturbances observed in Bscl2\/seipin-deficient mice","volume":"56","author":"Prieur","year":"2013","journal-title":"Diabetologia"},{"key":"10.1016\/j.omtm.2022.09.014_bib11","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1016\/j.molmet.2018.01.019","article-title":"Adipose specific disruption of seipin causes early-onset generalised lipodystrophy and altered fuel utilisation without severe metabolic disease","volume":"10","author":"Mcilroy","year":"2018","journal-title":"Mol. Metab."},{"key":"10.1016\/j.omtm.2022.09.014_bib12","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/j.bbrc.2015.02.147","article-title":"Expression of seipin in adipose tissue rescues lipodystrophy, hepatic steatosis and insulin resistance in seipin null mice","volume":"460","author":"Gao","year":"2015","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.omtm.2022.09.014_bib13","doi-asserted-by":"crossref","first-page":"E54","DOI":"10.1152\/ajpendo.00180.2018","article-title":"Adipose tissue transplantation ameliorates lipodystrophy-associated metabolic disorders in seipin-deficient mice","volume":"316","author":"Wang","year":"2019","journal-title":"Am. J. Physiol. Endocrinol. Metab."},{"key":"10.1016\/j.omtm.2022.09.014_bib14","doi-asserted-by":"crossref","first-page":"129781","DOI":"10.1172\/jci.insight.129781","article-title":"Targeting ATGL to rescue BSCL2 lipodystrophy and its associated cardiomyopathy","volume":"5","author":"Zhou","year":"2019","journal-title":"JCI Insight"},{"key":"10.1016\/j.omtm.2022.09.014_bib15","doi-asserted-by":"crossref","first-page":"653","DOI":"10.1038\/gt.2014.38","article-title":"Targeting adipose tissue via systemic gene therapy","volume":"21","author":"O'Neill","year":"2014","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2022.09.014_bib16","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.omtm.2017.06.002","article-title":"Targeting visceral fat by intraperitoneal delivery of novel AAV serotype vector restricting off-target transduction in liver","volume":"6","author":"Huang","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2022.09.014_bib17","doi-asserted-by":"crossref","first-page":"e8791","DOI":"10.15252\/emmm.201708791","article-title":"FGF21 gene therapy as treatment for obesity and insulin resistance","volume":"10","author":"Jimenez","year":"2018","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2022.09.014_bib18","doi-asserted-by":"crossref","first-page":"236","DOI":"10.1016\/j.omtm.2020.09.009","article-title":"Adipose tissue: an emerging target for adeno-associated viral vectors","volume":"19","author":"Bates","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2022.09.014_bib19","doi-asserted-by":"crossref","first-page":"2055","DOI":"10.2337\/db08-0184","article-title":"The human lipodystrophy gene BSCL2\/seipin may be essential for normal adipocyte differentiation","volume":"57","author":"Payne","year":"2008","journal-title":"Diabetes"},{"key":"10.1016\/j.omtm.2022.09.014_bib20","doi-asserted-by":"crossref","first-page":"4552","DOI":"10.1210\/en.2009-0236","article-title":"The human lipodystrophy gene product Berardinelli-Seip congenital lipodystrophy 2\/seipin plays a key role in adipocyte differentiation","volume":"150","author":"Chen","year":"2009","journal-title":"Endocrinology"},{"key":"10.1016\/j.omtm.2022.09.014_bib21","doi-asserted-by":"crossref","first-page":"4215","DOI":"10.1210\/en.2014-1292","article-title":"Molecular mechanisms underlying fasting modulated liver insulin sensitivity and metabolism in male lipodystrophic Bscl2\/Seipin-deficient mice","volume":"155","author":"Chen","year":"2014","journal-title":"Endocrinology"},{"key":"10.1016\/j.omtm.2022.09.014_bib22","doi-asserted-by":"crossref","first-page":"3410","DOI":"10.2337\/db16-0327","article-title":"FGF21 improves the adipocyte dysfunction related to seipin deficiency","volume":"65","author":"Dollet","year":"2016","journal-title":"Diabetes"},{"key":"10.1016\/j.omtm.2022.09.014_bib23","doi-asserted-by":"crossref","first-page":"428","DOI":"10.5152\/dir.2017.17019","article-title":"Determining residual adipose tissue characteristics with MRI in patients with various subtypes of lipodystrophy","volume":"23","author":"Altay","year":"2017","journal-title":"Diagn. Interv. Radiol."},{"key":"10.1016\/j.omtm.2022.09.014_bib24","doi-asserted-by":"crossref","first-page":"249","DOI":"10.2337\/db19-0447","article-title":"Clinical and molecular prevalence of lipodystrophy in an unascertained large clinical care cohort","volume":"69","author":"Gonzaga-Jauregui","year":"2020","journal-title":"Diabetes"},{"key":"10.1016\/j.omtm.2022.09.014_bib25","article-title":"BMP7 overexpression in adipose\u00a0tissue induces white adipogenesis and improves insulin sensitivity in ob\/ob mice","author":"Casana","year":"2020","journal-title":"Int. J. Obes"},{"key":"10.1016\/j.omtm.2022.09.014_bib26","doi-asserted-by":"crossref","first-page":"5550","DOI":"10.1096\/fj.201701427R","article-title":"Renal injury in Seipin-deficient lipodystrophic mice and its reversal by adipose tissue transplantation or leptin administration alone: adipose tissue-kidney crosstalk","volume":"32","author":"Liu","year":"2018","journal-title":"FASEB J"},{"key":"10.1016\/j.omtm.2022.09.014_bib27","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1016\/j.cell.2008.09.036","article-title":"Identification of white adipocyte progenitor cells in\u00a0vivo","volume":"135","author":"Rodeheffer","year":"2008","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2022.09.014_bib28","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1172\/JCI7901","article-title":"Surgical implantation of adipose tissue reverses diabetes in lipoatrophic mice","volume":"105","author":"Gavrilova","year":"2000","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2022.09.014_bib29","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1038\/nature06902","article-title":"Dynamics of fat cell turnover in humans","volume":"453","author":"Spalding","year":"2008","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2022.09.014_bib30","doi-asserted-by":"crossref","first-page":"e60361","DOI":"10.1371\/journal.pone.0060361","article-title":"Assessment of tropism and effectiveness of new primate-derived hybrid recombinant AAV serotypes in the mouse and primate retina","volume":"8","author":"Charbel Issa","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2022.09.014_bib31","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1038\/mtm.2013.8","article-title":"Adipose tissue insulin receptor knockdown via a new primate-derived hybrid recombinant AAV serotype","volume":"1","author":"Liu","year":"2014","journal-title":"Molecular Therapy - Methods & Clinical Development"},{"key":"10.1016\/j.omtm.2022.09.014_bib32","doi-asserted-by":"crossref","first-page":"376","DOI":"10.1038\/ncb3122","article-title":"Rapid depot-specific activation of adipocyte precursor cells at the onset of obesity","volume":"17","author":"Jeffery","year":"2015","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.omtm.2022.09.014_bib33","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1016\/j.cmet.2016.05.012","article-title":"The adipose tissue microenvironment regulates depot-specific adipogenesis in obesity","volume":"24","author":"Jeffery","year":"2016","journal-title":"Cell Metab."},{"key":"10.1016\/j.omtm.2022.09.014_bib34","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-018-36078-9","article-title":"Female adipose tissue-specific Bscl2 knockout mice develop only moderate metabolic dysfunction when housed at thermoneutrality and fed a high-fat diet","volume":"8","author":"Mcilroy","year":"2018","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2022.09.014_bib35","doi-asserted-by":"crossref","first-page":"dmm042655","DOI":"10.1242\/dmm.042655","article-title":"Ablation of Bscl2\/seipin in hepatocytes does not cause metabolic dysfunction in congenital generalised lipodystrophy","volume":"13","author":"Mcilroy","year":"2020","journal-title":"Dis. Model. Mech."},{"key":"10.1016\/j.omtm.2022.09.014_bib36","doi-asserted-by":"crossref","first-page":"e291","DOI":"10.1542\/peds.2006-3165","article-title":"Metabolic correction induced by leptin replacement treatment in young children with Berardinelli-Seip congenital lipoatrophy","volume":"120","author":"Beltrand","year":"2007","journal-title":"Pediatrics"},{"key":"10.1016\/j.omtm.2022.09.014_bib37","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1007\/s00125-009-1502-9","article-title":"Efficacy of leptin therapy in the different forms of human lipodystrophy","volume":"53","author":"Chong","year":"2010","journal-title":"Diabetologia"},{"key":"10.1016\/j.omtm.2022.09.014_bib38","doi-asserted-by":"crossref","first-page":"1083","DOI":"10.1530\/EJE-09-1027","article-title":"Resistance to leptin-replacement therapy in Berardinelli-Seip congenital lipodystrophy: an immunological origin","volume":"162","author":"Beltrand","year":"2010","journal-title":"Eur. J. Endocrinol."},{"key":"10.1016\/j.omtm.2022.09.014_bib39","doi-asserted-by":"crossref","first-page":"368","DOI":"10.1016\/j.cmet.2014.06.003","article-title":"Bone marrow adipose tissue is an endocrine organ that contributes to increased circulating adiponectin during caloric restriction","volume":"20","author":"Cawthorn","year":"2014","journal-title":"Cell Metab."},{"key":"10.1016\/j.omtm.2022.09.014_bib40","doi-asserted-by":"crossref","first-page":"70","DOI":"10.1016\/j.mce.2019.03.005","article-title":"SEIPIN overexpression in the liver may alleviate hepatic steatosis by influencing the intracellular calcium level","volume":"488","author":"Li","year":"2019","journal-title":"Mol. Cell. Endocrinol."},{"key":"10.1016\/j.omtm.2022.09.014_bib41","doi-asserted-by":"crossref","first-page":"441","DOI":"10.3390\/jcm10030441","article-title":"Bscl2 deficiency does not directly impair the innate immune response in a murine model of generalized lipodystrophy","volume":"10","author":"Roumane","year":"2021","journal-title":"J.\u00a0Clin. Med."},{"key":"10.1016\/j.omtm.2022.09.014_bib42","doi-asserted-by":"crossref","first-page":"2402","DOI":"10.1210\/jcem.85.7.6661","article-title":"Quantitative insulin sensitivity check index: a simple, accurate method for assessing insulin sensitivity in humans","volume":"85","author":"Katz","year":"2000","journal-title":"J.\u00a0Clin. Endocrinol. Metab."}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050122001395?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050122001395?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:27:48Z","timestamp":1762421268000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050122001395"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,12]]},"references-count":42,"alternative-id":["S2329050122001395"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2022.09.014","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2022,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Gene therapy restores adipose tissue and metabolic health in a pre-clinical mouse model of lipodystrophy","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2022.09.014","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 The Authors.","name":"copyright","label":"Copyright"}]}}