{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,23]],"date-time":"2026-02-23T10:47:07Z","timestamp":1771843627922,"version":"3.50.1"},"reference-count":43,"publisher":"Elsevier BV","issue":"9","license":[{"start":{"date-parts":[[2010,9,1]],"date-time":"2010-09-01T00:00:00Z","timestamp":1283299200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,10,7]],"date-time":"2016-10-07T00:00:00Z","timestamp":1475798400000},"content-version":"vor","delay-in-days":2228,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2010,9]]},"DOI":"10.1038\/mt.2010.102","type":"journal-article","created":{"date-parts":[[2010,6,22]],"date-time":"2010-06-22T14:13:19Z","timestamp":1277215999000},"page":"1624-1632","source":"Crossref","is-referenced-by-count":41,"title":["Efficient KRT14 Targeting and Functional Characterization of Transplanted Human Keratinocytes for the Treatment of Epidermolysis Bullosa Simplex"],"prefix":"10.1016","volume":"18","author":[{"given":"Lisa M","family":"Petek","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Philip","family":"Fleckman","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Daniel G","family":"Miller","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1038\/mt.2010.102_bib1","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1002\/ajmg.c.30035","article-title":"Progress in epidermolysis bullosa: genetic classification and clinical implications","volume":"131C","author":"Uitto","year":"2004","journal-title":"Am J Med Genet C Semin Med Genet"},{"key":"10.1038\/mt.2010.102_bib2","doi-asserted-by":"crossref","first-page":"931","DOI":"10.1016\/j.jaad.2008.02.004","article-title":"The classification of inherited epidermolysis bullosa (EB): Report of the Third International Consensus Meeting on Diagnosis and Classification of EB","volume":"58","author":"Fine","year":"2008","journal-title":"J Am Acad Dermatol"},{"key":"10.1038\/mt.2010.102_bib3","doi-asserted-by":"crossref","first-page":"1202","DOI":"10.1126\/science.1720261","article-title":"Epidermolysis bullosa simplex: evidence in two families for keratin gene abnormalities","volume":"254","author":"Bonifas","year":"1991","journal-title":"Science"},{"key":"10.1038\/mt.2010.102_bib4","doi-asserted-by":"crossref","first-page":"1301","DOI":"10.1016\/0092-8674(91)90051-Y","article-title":"Point mutations in human keratin 14 genes of epidermolysis bullosa simplex patients: genetic and functional analyses","volume":"66","author":"Coulombe","year":"1991","journal-title":"Cell"},{"key":"10.1038\/mt.2010.102_bib5","doi-asserted-by":"crossref","first-page":"970","DOI":"10.1046\/j.1523-1747.2001.01324.x","article-title":"Expression of a truncated keratin 5 may contribute to severe palmar\u2013plantar hyperkeratosis in epidermolysis bullosa simplex patients","volume":"116","author":"Livingston","year":"2001","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2010.102_bib6","doi-asserted-by":"crossref","first-page":"877","DOI":"10.1111\/1523-1747.ep12607050","article-title":"A common keratin 5 gene mutation in epidermolysis bullosa simplex\u2013Weber-Cockayne","volume":"104","author":"Ehrlich","year":"1995","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2010.102_bib7","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1111\/1523-1747.ep12365079","article-title":"A keratin 14 mutational hot spot for epidermolysis bullosa simplex, Dowling-Meara: implications for diagnosis","volume":"101","author":"Stephens","year":"1993","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2010.102_bib8","series-title":"Epidermolysis Bullosa Simplex. In: GeneReviews at Gene Tests: Medical Genetics Information Resource (database online)","first-page":"1997","author":"Pfender","year":"2008"},{"key":"10.1038\/mt.2010.102_bib9","doi-asserted-by":"crossref","first-page":"1351","DOI":"10.1038\/nm1328","article-title":"Stem cells in the hair follicle bulge contribute to wound repair but not to homeostasis of the epidermis","volume":"11","author":"Ito","year":"2005","journal-title":"Nat Med"},{"key":"10.1038\/mt.2010.102_bib10","doi-asserted-by":"crossref","first-page":"713","DOI":"10.1016\/0092-8674(93)90251-K","article-title":"Separation of human epidermal stem cells from transit amplifying cells on the basis of differences in integrin function and expression","volume":"73","author":"Jones","year":"1993","journal-title":"Cell"},{"key":"10.1038\/mt.2010.102_bib11","doi-asserted-by":"crossref","first-page":"448","DOI":"10.1056\/NEJM198408163110706","article-title":"Permanent coverage of large burn wounds with autologous cultured human epithelium","volume":"311","author":"Gallico 3rd","year":"1984","journal-title":"N Engl J Med"},{"key":"10.1038\/mt.2010.102_bib12","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/S0092-8674(75)80001-8","article-title":"Serial cultivation of strains of human epidermal keratinocytes: the formation of keratinizing colonies from single cells","volume":"6","author":"Rheinwald","year":"1975","journal-title":"Cell"},{"key":"10.1038\/mt.2010.102_bib13","doi-asserted-by":"crossref","first-page":"2302","DOI":"10.1073\/pnas.84.8.2302","article-title":"Three clonal types of keratinocyte with different capacities for multiplication","volume":"84","author":"Barrandon","year":"1987","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2010.102_bib14","doi-asserted-by":"crossref","first-page":"10371","DOI":"10.1073\/pnas.93.19.10371","article-title":"Clonal analysis of stably transduced human epidermal stem cells in culture","volume":"93","author":"Mathor","year":"1996","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2010.102_bib15","doi-asserted-by":"crossref","first-page":"1397","DOI":"10.1038\/nm1504","article-title":"Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells","volume":"12","author":"Mavilio","year":"2006","journal-title":"Nat Med"},{"key":"10.1038\/mt.2010.102_bib16","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1016\/S0305-4179(01)00107-3","article-title":"Allogeneic skin substitutes applied to burns patients","volume":"28","author":"Nanchahal","year":"2002","journal-title":"Burns"},{"key":"10.1038\/mt.2010.102_bib17","doi-asserted-by":"crossref","first-page":"528","DOI":"10.1007\/s00403-005-0547-y","article-title":"Recombinant adeno-associated virus type 2-mediated gene transfer into human keratinocytes is influenced by both the ubiquitin\/proteasome pathway and epidermal growth factor receptor tyrosine kinase","volume":"296","author":"Braun-Falco","year":"2005","journal-title":"Arch Dermatol Res"},{"key":"10.1038\/mt.2010.102_bib18","doi-asserted-by":"crossref","first-page":"1178","DOI":"10.1002\/jgm.768","article-title":"Assessment of optimal transduction of primary human skin keratinocytes by viral vectors","volume":"7","author":"Gagnoux-Palacios","year":"2005","journal-title":"J Gene Med"},{"key":"10.1038\/mt.2010.102_bib19","doi-asserted-by":"crossref","first-page":"767","DOI":"10.1038\/ng1380","article-title":"Adeno-associated virus vectors integrate at chromosome breakage sites","volume":"36","author":"Miller","year":"2004","journal-title":"Nat Genet"},{"key":"10.1038\/mt.2010.102_bib20","doi-asserted-by":"crossref","first-page":"325","DOI":"10.1038\/ng0498-325","article-title":"Human gene targeting by viral vectors","volume":"18","author":"Russell","year":"1998","journal-title":"Nat Genet"},{"key":"10.1038\/mt.2010.102_bib21","doi-asserted-by":"crossref","first-page":"735","DOI":"10.1038\/nbt0702-735","article-title":"Targeted transgene insertion into human chromosomes by adeno-associated virus vectors","volume":"20","author":"Hirata","year":"2002","journal-title":"Nat Biotechnol"},{"key":"10.1038\/mt.2010.102_bib22","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1126\/science.2497519","article-title":"Mouse embryonic stem cells and reporter constructs to detect developmentally regulated genes","volume":"244","author":"Gossler","year":"1989","journal-title":"Science"},{"key":"10.1038\/mt.2010.102_bib23","doi-asserted-by":"crossref","first-page":"828","DOI":"10.1038\/nm1085","article-title":"Systemic delivery of genes to striated muscles using adeno-associated viral vectors","volume":"10","author":"Gregorevic","year":"2004","journal-title":"Nat Med"},{"key":"10.1038\/mt.2010.102_bib24","doi-asserted-by":"crossref","first-page":"907","DOI":"10.1089\/hum.2008.061","article-title":"Human gene targeting favors insertions over deletions","volume":"19","author":"Russell","year":"2008","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2010.102_bib25","doi-asserted-by":"crossref","first-page":"1963","DOI":"10.1128\/JVI.62.6.1963-1973.1988","article-title":"Adeno-associated virus general transduction vectors: analysis of proviral structures","volume":"62","author":"McLaughlin","year":"1988","journal-title":"J Virol"},{"key":"10.1038\/mt.2010.102_bib26","doi-asserted-by":"crossref","first-page":"9451","DOI":"10.1128\/JVI.74.20.9451-9463.2000","article-title":"Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo","volume":"74","author":"Nakai","year":"2000","journal-title":"J Virol"},{"key":"10.1038\/mt.2010.102_bib27","doi-asserted-by":"crossref","first-page":"1529","DOI":"10.1096\/fj.01-0082com","article-title":"A cutaneous gene therapy approach to human leptin deficiencies: correction of the murine ob\/ob phenotype using leptin-targeted keratinocyte grafts","volume":"15","author":"Larcher","year":"2001","journal-title":"FASEB J"},{"key":"10.1038\/mt.2010.102_bib28","doi-asserted-by":"crossref","first-page":"1670","DOI":"10.1038\/sj.mt.6300238","article-title":"Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapy","volume":"15","author":"Larcher","year":"2007","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.102_bib29","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1016\/j.cell.2007.02.045","article-title":"p63 Is essential for the proliferative potential of stem cells in stratified epithelia","volume":"129","author":"Senoo","year":"2007","journal-title":"Cell"},{"key":"10.1038\/mt.2010.102_bib30","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1111\/1523-1747.ep12475646","article-title":"New techniques for the grafting of cultured human epidermal cells onto athymic animals","volume":"91","author":"Barrandon","year":"1988","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2010.102_bib31","doi-asserted-by":"crossref","first-page":"1588","DOI":"10.1097\/00007890-200012150-00009","article-title":"Long-term regeneration of human epidermis on third degree burns transplanted with autologous cultured epithelium grown on a fibrin matrix","volume":"70","author":"Ronfard","year":"2000","journal-title":"Transplantation"},{"key":"10.1038\/mt.2010.102_bib32","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1146\/annurev.cellbio.22.010305.104357","article-title":"Epidermal stem cells of the skin","volume":"22","author":"Blanpain","year":"2006","journal-title":"Annu Rev Cell Dev Biol"},{"key":"10.1038\/mt.2010.102_bib33","doi-asserted-by":"crossref","first-page":"1276","DOI":"10.1038\/nbt.1503","article-title":"Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes","volume":"26","author":"Aasen","year":"2008","journal-title":"Nat Biotechnol"},{"key":"10.1038\/mt.2010.102_bib34","doi-asserted-by":"crossref","first-page":"646","DOI":"10.1161\/01.HYP.22.4.646","article-title":"Double knockouts. Production of mutant cell lines in cardiovascular research","volume":"22","author":"Mortensen","year":"1993","journal-title":"Hypertension"},{"key":"10.1038\/mt.2010.102_bib35","doi-asserted-by":"crossref","first-page":"7036","DOI":"10.1073\/pnas.88.16.7036","article-title":"Embryonic stem cells lacking a functional inhibitory G-protein subunit (alpha i2) produced by gene targeting of both alleles","volume":"88","author":"Mortensen","year":"1991","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2010.102_bib36","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1038\/348649a0","article-title":"Consecutive inactivation of both alleles of the pim-1 proto-oncogene by homologous recombination in embryonic stem cells","volume":"348","author":"te Riele","year":"1990","journal-title":"Nature"},{"key":"10.1038\/mt.2010.102_bib37","doi-asserted-by":"crossref","first-page":"2186","DOI":"10.1038\/sj.mt.6300292","article-title":"Safe selection of genetically manipulated human primary keratinocytes with very high growth potential using CD24","volume":"15","author":"Bergoglio","year":"2007","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.102_bib38","doi-asserted-by":"crossref","first-page":"677","DOI":"10.1016\/0092-8674(85)90216-8","article-title":"Enzymatic cross-linking of involucrin and other proteins by keratinocyte particulates in vitro","volume":"40","author":"Simon","year":"1985","journal-title":"Cell"},{"key":"10.1038\/mt.2010.102_bib39","doi-asserted-by":"crossref","first-page":"3096","DOI":"10.1128\/JVI.61.10.3096-3101.1987","article-title":"A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication","volume":"61","author":"Samulski","year":"1987","journal-title":"J Virol"},{"key":"10.1038\/mt.2010.102_bib40","doi-asserted-by":"crossref","first-page":"2745","DOI":"10.1089\/hum.1998.9.18-2745","article-title":"Novel tools for production and purification of recombinant adenoassociated virus vectors","volume":"9","author":"Grimm","year":"1998","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2010.102_bib41","doi-asserted-by":"crossref","first-page":"1427","DOI":"10.1038\/mt.2009.116","article-title":"Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature","volume":"17","author":"Gregorevic","year":"2009","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.102_bib42","doi-asserted-by":"crossref","first-page":"959","DOI":"10.1089\/10430340252939069","article-title":"A preclinical model for the analysis of genetically modified human skin in vivo","volume":"13","author":"Del Rio","year":"2002","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2010.102_bib43","doi-asserted-by":"crossref","first-page":"621","DOI":"10.1016\/S0305-4179(98)00107-7","article-title":"Large surface of cultured human epithelium obtained on a dermal matrix based on live fibroblast-containing fibrin gels","volume":"24","author":"Meana","year":"1998","journal-title":"Burns"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616308383?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616308383?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,6,6]],"date-time":"2020-06-06T01:01:53Z","timestamp":1591405313000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616308383"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2010,9]]},"references-count":43,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2010,9]]}},"alternative-id":["S1525001616308383"],"URL":"https:\/\/doi.org\/10.1038\/mt.2010.102","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2010,9]]}}}