{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T16:01:32Z","timestamp":1791388892528,"version":"4.3.3"},"reference-count":79,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2019,6,1]],"date-time":"2019-06-01T00:00:00Z","timestamp":1559347200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,6,1]],"date-time":"2019-06-01T00:00:00Z","timestamp":1559347200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2020,6,1]],"date-time":"2020-06-01T00:00:00Z","timestamp":1590969600000},"content-version":"vor","delay-in-days":366,"URL":"http:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"}],"funder":[{"name":"Office of Research, Palo Alto Veterans Affairs Medical Center"},{"DOI":"10.13039\/100002077","name":"Epidermolysis Bullosa Medical Research Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002077","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Epidermolysis Bullosa Research Partnership"},{"DOI":"10.13039\/501100011960","name":"DEBRA International","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100011960","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R13-AR009431-52"],"award-info":[{"award-number":["R13-AR009431-52"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["jidonline.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Investigative Dermatology"],"published-print":{"date-parts":[[2019,6]]},"DOI":"10.1016\/j.jid.2018.11.036","type":"journal-article","created":{"date-parts":[[2019,5,7]],"date-time":"2019-05-07T11:32:00Z","timestamp":1557228720000},"page":"1221-1226","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":82,"title":["Gene Therapy for Epidermolysis Bullosa"],"prefix":"10.1016","volume":"139","author":[{"given":"M. Peter","family":"Marinkovich","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5510-6502","authenticated-orcid":false,"given":"Jean Y.","family":"Tang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jid.2018.11.036_bib1","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1056\/NEJMoa0805817","article-title":"Gene therapy for immunodeficiency due to adenosine deaminase deficiency","volume":"360","author":"Aiuti","year":"2009","journal-title":"N\u00a0Engl J Med"},{"key":"10.1016\/j.jid.2018.11.036_bib2","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":"10.1016\/j.jid.2018.11.036_bib3","doi-asserted-by":"crossref","first-page":"778","DOI":"10.1016\/j.jid.2016.10.038","article-title":"Closure of a large chronic wound through transplantation of gene-corrected epidermal stem cells","volume":"137","author":"Bauer","year":"2017","journal-title":"J\u00a0Invest Dermatol"},{"issue":"5035","key":"10.1016\/j.jid.2018.11.036_bib4","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":"10.1016\/j.jid.2018.11.036_bib5","first-page":"821","article-title":"Cultured skin substitutes reduce requirements for harvesting of skin autograft for closure of excised, full-thickness burns","volume":"60","author":"Boyce","year":"2006","journal-title":"J\u00a0Trauma"},{"key":"10.1016\/j.jid.2018.11.036_bib6","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1111\/1523-1747.ep12277331","article-title":"Anchoring fibrils and type VII collagen are absent from skin in severe recessive dystrophic epidermolysis bullosa","volume":"93","author":"Bruckner-Tuderman","year":"1989","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib7","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1111\/j.1749-6632.1985.tb51156.x","article-title":"The structure of type VII collagen","volume":"460","author":"Burgeson","year":"1985","journal-title":"Ann NY Acad Sci"},{"issue":"2 Pt. 1","key":"10.1016\/j.jid.2018.11.036_bib8","doi-asserted-by":"crossref","first-page":"246","DOI":"10.1016\/S0190-9622(87)70199-6","article-title":"Treatment of junctional epidermolysis bullosa with epidermal autografts","volume":"17","author":"Carter","year":"1987","journal-title":"J\u00a0Am Acad Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib9","doi-asserted-by":"crossref","first-page":"177","DOI":"10.1046\/j.1523-1747.1999.00491.x","article-title":"NC1 domain of type VII collagen binds to the \u03b23 chain of laminin 5 via a unique subdomain within the fibronectin-like repeats","volume":"112","author":"Chen","year":"1999","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib10","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1038\/ng0593-62","article-title":"A\u00a0missense mutation in type VII collagen in two affected siblings with recessive dystrophic epidermolysis bullosa","volume":"4","author":"Christiano","year":"1993","journal-title":"Nat Genet"},{"issue":"32","key":"10.1016\/j.jid.2018.11.036_bib11","doi-asserted-by":"crossref","first-page":"20256","DOI":"10.1016\/S0021-9258(17)31984-1","article-title":"Cloning of human type VII collagen. Complete primary sequence of the \u03b11(VII) chain and identification of intragenic polymorphisms","volume":"269","author":"Christiano","year":"1994","journal-title":"J\u00a0Biol Chem"},{"key":"10.1016\/j.jid.2018.11.036_bib12","doi-asserted-by":"crossref","first-page":"429","DOI":"10.3109\/14653241003587637","article-title":"Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa","volume":"12","author":"Conget","year":"2010","journal-title":"Cytotherapy"},{"key":"10.1016\/j.jid.2018.11.036_bib13","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":"10.1016\/j.jid.2018.11.036_bib14","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.stemcr.2013.11.001","article-title":"Long-term stability and safety of transgenic cultured epidermal stem cells in gene therapy of junctional epidermolysis bullosa","volume":"2","author":"De Rosa","year":"2014","journal-title":"Stem Cell Reports"},{"key":"10.1016\/j.jid.2018.11.036_bib15","doi-asserted-by":"crossref","first-page":"1628","DOI":"10.1172\/JCI116033","article-title":"Epiligrin, the major human keratinocyte integrin ligand, is a target in both an acquired autoimmune and an inherited subepidermal blistering skin disease","volume":"90","author":"Domloge-Hultsch","year":"1992","journal-title":"J\u00a0Clin Invest"},{"key":"10.1016\/j.jid.2018.11.036_bib16","doi-asserted-by":"crossref","first-page":"96","DOI":"10.1111\/dth.12305","article-title":"Treatment of dystrophic epidermolysis bullosa with bone marrow non-hematopoeitic stem cells: a randomized controlled trial","volume":"29","author":"El-Darouti","year":"2016","journal-title":"Dermatol Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib17","doi-asserted-by":"crossref","first-page":"1225","DOI":"10.1001\/archderm.136.10.1225","article-title":"Tissue-engineered skin (Apligraf) in the healing of patients with epidermolysis bullosa wounds","volume":"136","author":"Falabella","year":"2000","journal-title":"Arch Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib18","doi-asserted-by":"crossref","first-page":"886","DOI":"10.1067\/mjd.2003.502","article-title":"Graftskin therapy in epidermolysis bullosa","volume":"48","author":"Fivenson","year":"2003","journal-title":"J\u00a0Am Acad Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib19","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1111\/bjd.13737","article-title":"Characterization of patients with dystrophic epidermolysis bullosa for collagen VII therapy","volume":"173","author":"Gorell","year":"2015","journal-title":"Br J Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib20","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1056\/NEJM200301163480314","article-title":"A\u00a0serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency","volume":"348","author":"Hacein-Bey-Abina","year":"2003","journal-title":"N\u00a0Engl J Med"},{"issue":"5644","key":"10.1016\/j.jid.2018.11.036_bib21","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1126\/science.1088547","article-title":"LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1","volume":"302","author":"Hacein-Bey-Abina","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.jid.2018.11.036_bib22","doi-asserted-by":"crossref","first-page":"2150","DOI":"10.1016\/j.jid.2016.06.609","article-title":"Genotype, clinical course, and therapeutic decision making in 76 infants with severe generalized junctional epidermolysis bullosa","volume":"136","author":"Hammersen","year":"2016","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib23","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1016\/j.matbio.2018.04.001","article-title":"Epidermolysis bullosa: molecular pathology of connective tissue components in the cutaneous basement membrane zone","volume":"71\u201372","author":"Has","year":"2018","journal-title":"Matrix Biol"},{"key":"10.1016\/j.jid.2018.11.036_bib24","unstructured":"Hebra FV. Arztlicher Bericht des K.K allegemeinen Krankenhauses zu Wien vom Jare 1870. Vienna, Austria; 1870."},{"key":"10.1016\/j.jid.2018.11.036_bib25","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1038\/ng1193-287","article-title":"A\u00a0homozygous insertion-deletion in the type VII collagen gene (COL7A1) in Hallopeau-Siemens dystrophic epidermolysis bullosa","volume":"5","author":"Hilal","year":"1993","journal-title":"Nat Genet"},{"issue":"7680","key":"10.1016\/j.jid.2018.11.036_bib26","doi-asserted-by":"crossref","first-page":"327","DOI":"10.1038\/nature24487","article-title":"Regeneration of the entire human epidermis using transgenic stem cells","volume":"551","author":"Hirsch","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.jid.2018.11.036_bib27","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1016\/S0923-1811(03)00158-0","article-title":"Usefulness of immunoblotting using purified laminin 5 in the diagnosis of anti-laminin 5 cicatricial pemphigoid","volume":"33","author":"Hisamatsu","year":"2003","journal-title":"J\u00a0Dermatol Sci"},{"key":"10.1016\/j.jid.2018.11.036_bib28","doi-asserted-by":"crossref","first-page":"1346","DOI":"10.1016\/j.jid.2016.02.811","article-title":"Gene-corrected fibroblast therapy for recessive dystrophic epidermolysis bullosa using a self-inactivating COL7A1 retroviral vector","volume":"136","author":"Jackow","year":"2016","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib29","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1083\/jcb.104.3.611","article-title":"Type VII collagen forms an extended network of anchoring fibrils","volume":"104","author":"Keene","year":"1987","journal-title":"J\u00a0Cell Biol"},{"key":"10.1016\/j.jid.2018.11.036_bib30","doi-asserted-by":"crossref","first-page":"1116","DOI":"10.1016\/j.jid.2016.02.002","article-title":"Collagen VII half-life at the dermal-epidermal junction zone: implications for mechanisms and therapy of genodermatoses","volume":"136","author":"Kuhl","year":"2016","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib31","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1016\/j.det.2009.10.006","article-title":"Herlitz junctional epidermolysis bullosa","volume":"28","author":"Laimer","year":"2010","journal-title":"Dermatol Clin"},{"key":"10.1016\/j.jid.2018.11.036_bib32","doi-asserted-by":"crossref","first-page":"1831","DOI":"10.1172\/JCI116774","article-title":"Epitope mapping of type VII collagen. Identification of discrete peptide sequences recognized by sera from patients with acquired epidermolysis bullosa","volume":"92","author":"Lapiere","year":"1993","journal-title":"J\u00a0Clin Invest"},{"key":"10.1016\/j.jid.2018.11.036_bib33","doi-asserted-by":"crossref","first-page":"1509","DOI":"10.1172\/JCI118942","article-title":"Passive transfer of anti-laminin 5 antibodies induces subepidermal blisters in neonatal mice","volume":"98","author":"Lazarova","year":"1996","journal-title":"J\u00a0Clin Invest"},{"issue":"5 Suppl","key":"10.1016\/j.jid.2018.11.036_bib34","doi-asserted-by":"crossref","first-page":"S100","DOI":"10.1016\/j.jid.2018.03.599","article-title":"A\u00a0phase 1\/2 study of genetically-corrected, collagen VII expressing autologous human dermal fibroblasts injected into the skin of patients with recessive dystrophic epidermolysis bullosa (RDEB)","volume":"138","author":"Marinkovich","year":"2018","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib35","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1002\/aja.1001970404","article-title":"Cellular origin of the dermal-epidermal basement membrane","volume":"197","author":"Marinkovich","year":"1993","journal-title":"Dev Dyn"},{"key":"10.1016\/j.jid.2018.11.036_bib36","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1083\/jcb.119.3.695","article-title":"The dermal-epidermal junction of human skin contains a novel laminin variant","volume":"119","author":"Marinkovich","year":"1992","journal-title":"J\u00a0Cell Biol"},{"key":"10.1016\/j.jid.2018.11.036_bib37","first-page":"295","article-title":"Basement membrane proteins kalinin and nicein are structurally and immunologically identical","volume":"69","author":"Marinkovich","year":"1993","journal-title":"Lab Invest"},{"key":"10.1016\/j.jid.2018.11.036_bib38","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":"10.1016\/j.jid.2018.11.036_bib39","doi-asserted-by":"crossref","first-page":"658","DOI":"10.5021\/ad.2015.27.6.658","article-title":"Recently identified forms of epidermolysis bullosa","volume":"27","author":"McGrath","year":"2015","journal-title":"Ann Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib40","doi-asserted-by":"crossref","first-page":"725","DOI":"10.1038\/mt.2013.290","article-title":"Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant","volume":"22","author":"Melo","year":"2014","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib41","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1111\/j.1600-0625.1992.tb00080.x","article-title":"Kalinin is abnormally expressed in epithelial basement membranes of Herlitz\u2019s junctional epidermolysis bullosa patients","volume":"1","author":"Meneguzzi","year":"1992","journal-title":"Exp Dermatol"},{"issue":"5716","key":"10.1016\/j.jid.2018.11.036_bib42","doi-asserted-by":"crossref","first-page":"1773","DOI":"10.1126\/science.1106209","article-title":"Type VII collagen is required for Ras-driven human epidermal tumorigenesis","volume":"307","author":"Ortiz-Urda","year":"2005","journal-title":"Science"},{"key":"10.1016\/j.jid.2018.11.036_bib43","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1172\/JCI200317193","article-title":"Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue","volume":"111","author":"Ortiz-Urda","year":"2003","journal-title":"J\u00a0Clin Invest"},{"key":"10.1016\/j.jid.2018.11.036_bib44","doi-asserted-by":"crossref","first-page":"1099","DOI":"10.1038\/sj.gt.3301978","article-title":"Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer","volume":"10","author":"Ortiz-Urda","year":"2003","journal-title":"Gene Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib45","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1089\/104303403765701204","article-title":"\u03c6C31 integrase-mediated nonviral genetic correction of junctional epidermolysis bullosa","volume":"14","author":"Ortiz-Urda","year":"2003","journal-title":"Hum Gene Ther"},{"issue":"4","key":"10.1016\/j.jid.2018.11.036_bib46","doi-asserted-by":"crossref","first-page":"946","DOI":"10.3390\/ijms19040946","article-title":"CRISPR\/Cas9-based cellular engineering for targeted gene overexpression","volume":"19","author":"Osborn","year":"2018","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.jid.2018.11.036_bib47","doi-asserted-by":"crossref","first-page":"1151","DOI":"10.1038\/mt.2013.56","article-title":"TALEN-based gene correction for epidermolysis bullosa","volume":"21","author":"Osborn","year":"2013","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib48","first-page":"263","article-title":"A\u00a0special fibril of the dermis","volume":"27","author":"Palade","year":"1966","journal-title":"J\u00a0Cell Biol"},{"key":"10.1016\/j.jid.2018.11.036_bib49","first-page":"119","article-title":"Human type VII collagen: cDNA cloning and chromosomal mapping of the gene (COL7A1) on chromosome 3 to dominant dystrophic epidermolysis bullosa","volume":"24","author":"Parente","year":"1991","journal-title":"Am J Hum Genet"},{"key":"10.1016\/j.jid.2018.11.036_bib50","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1038\/jid.1962.156","article-title":"Studies on the pathogenesis of epidermolysis bullosa","volume":"39","author":"Pearson","year":"1962","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib51","doi-asserted-by":"crossref","first-page":"930","DOI":"10.1038\/gt.2010.36","article-title":"Immune reactivity to type VII collagen: implications for gene therapy of recessive dystrophic epidermolysis bullosa","volume":"17","author":"Pendaries","year":"2010","journal-title":"Gene Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib52","doi-asserted-by":"crossref","first-page":"2319","DOI":"10.1038\/jid.2015.158","article-title":"Potential of systemic allogeneic mesenchymal stromal cell therapy for children with recessive dystrophic epidermolysis bullosa","volume":"135","author":"Petrof","year":"2015","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib53","doi-asserted-by":"crossref","first-page":"1025","DOI":"10.1111\/bjd.12599","article-title":"Fibroblast cell therapy enhances initial healing in recessive dystrophic epidermolysis bullosa wounds: results of a randomized, vehicle-controlled trial","volume":"169","author":"Petrof","year":"2013","journal-title":"Br J Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib54","doi-asserted-by":"crossref","first-page":"441","DOI":"10.1097\/01.SAP.0000048065.92291.CD","article-title":"Surgical treatment of recessive dystrophic epidermolysis bullosa in the hand: use of tissue-engineered skin (Apligraf)","volume":"50","author":"Phillips","year":"2003","journal-title":"Ann Plast Surg"},{"key":"10.1016\/j.jid.2018.11.036_bib55","doi-asserted-by":"crossref","first-page":"526","DOI":"10.1038\/jid.2013.328","article-title":"TSG-6 released from intradermally injected mesenchymal stem cells accelerates wound healing and reduces tissue fibrosis in murine full-thickness skin wounds","volume":"134","author":"Qi","year":"2014","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib56","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1186\/s40425-016-0158-5","article-title":"Into the clinic: Talimogene laherparepvec (T-VEC), a first-in-class intratumoral oncolytic viral therapy","volume":"4","author":"Rehman","year":"2016","journal-title":"J\u00a0Immunother Cancer"},{"key":"10.1016\/j.jid.2018.11.036_bib57","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1038\/mt.2008.234","article-title":"Injection of recombinant human type VII collagen corrects the disease phenotype in a murine model of dystrophic epidermolysis bullosa","volume":"17","author":"Remington","year":"2009","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib58","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1016\/j.stem.2007.11.014","article-title":"Mesenchymal stem cell-mediated immunosuppression occurs via concerted action of chemokines and nitric oxide","volume":"2","author":"Ren","year":"2008","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.jid.2018.11.036_bib59","doi-asserted-by":"crossref","first-page":"1443","DOI":"10.1089\/104303401750298599","article-title":"Impact of laminin 5 \u03b23 gene versus protein replacement on gene expression patterns in junctional epidermolysis bullosa","volume":"12","author":"Robbins","year":"2001","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib60","doi-asserted-by":"crossref","first-page":"719","DOI":"10.1083\/jcb.138.3.719","article-title":"Laminin 5 binds the NC-1 domain of type VII collagen","volume":"138","author":"Rousselle","year":"1997","journal-title":"J\u00a0Cell Biol"},{"key":"10.1016\/j.jid.2018.11.036_bib61","doi-asserted-by":"crossref","first-page":"567","DOI":"10.1083\/jcb.114.3.567","article-title":"Kalinin: an epithelium-specific basement membrane adhesion molecule that is a component of anchoring filaments","volume":"114","author":"Rousselle","year":"1991","journal-title":"J\u00a0Cell Biol"},{"issue":"264","key":"10.1016\/j.jid.2018.11.036_bib62","doi-asserted-by":"crossref","first-page":"264ra163","DOI":"10.1126\/scitranslmed.3009540","article-title":"Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa","volume":"6","author":"Sebastiano","year":"2014","journal-title":"Sci Transl Med"},{"key":"10.1016\/j.jid.2018.11.036_bib63","doi-asserted-by":"crossref","first-page":"42","DOI":"10.1038\/sj.gt.3300809","article-title":"BP180 gene delivery in junctional epidermolysis bullosa","volume":"6","author":"Seitz","year":"1999","journal-title":"Gene Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib64","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1111\/j.1365-2133.1994.tb08550.x","article-title":"The basement membrane zone of the nail","volume":"131","author":"Sinclair","year":"1994","journal-title":"Br J Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib65","doi-asserted-by":"crossref","first-page":"1299","DOI":"10.1089\/hum.2010.023","article-title":"Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue","volume":"21","author":"Siprashvili","year":"2010","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jid.2018.11.036_bib66","doi-asserted-by":"crossref","first-page":"1808","DOI":"10.1001\/jama.2016.15588","article-title":"Safety and wound outcomes following genetically corrected autologous epidermal grafts in patients with recessive dystrophic epidermolysis bullosa","volume":"316","author":"Siprashvili","year":"2016","journal-title":"JAMA"},{"issue":"5 Suppl. 1","key":"10.1016\/j.jid.2018.11.036_bib67","doi-asserted-by":"crossref","first-page":"S89","DOI":"10.1016\/j.jid.2017.02.539","article-title":"Phase I\/IIa clinical trial for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes","volume":"137","author":"Siprashvili","year":"2017","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib68","doi-asserted-by":"crossref","first-page":"6609","DOI":"10.1073\/pnas.1016753108","article-title":"PDGFR\u03b1-positive cells in bone marrow are mobilized by high mobility group box 1 (HMGB1) to regenerate injured epithelia","volume":"108","author":"Tamai","year":"2011","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.jid.2018.11.036_bib69","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1186\/1750-1172-8-132","article-title":"Prevalence of specific anti-skin autoantibodies in a cohort of patients with inherited epidermolysis bullosa","volume":"8","author":"Tampoia","year":"2013","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.jid.2018.11.036_bib70","doi-asserted-by":"crossref","first-page":"1246","DOI":"10.1038\/jid.2013.523","article-title":"Patient-specific naturally gene-reverted induced pluripotent stem cells in recessive dystrophic epidermolysis bullosa","volume":"134","author":"Tolar","year":"2014","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib71","doi-asserted-by":"crossref","first-page":"2387","DOI":"10.1016\/j.jid.2016.07.029","article-title":"Targeted exon skipping restores type VII collagen expression and anchoring fibril formation in an in\u00a0vivo RDEB model","volume":"136","author":"Turczynski","year":"2016","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib72","series-title":"Proceedings of the Seventh International Symposium on Basement Membranes","first-page":"257","article-title":"Molecular basis of the junctional forms of epidermolysis bullosa, a disorder of the cutaneous basement membrane zone","author":"Uitto","year":"1995"},{"key":"10.1016\/j.jid.2018.11.036_bib73","doi-asserted-by":"crossref","first-page":"898","DOI":"10.1016\/j.jaad.2013.08.014","article-title":"A\u00a0phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa","volume":"69","author":"Venugopal","year":"2013","journal-title":"J\u00a0Am Acad Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib74","doi-asserted-by":"crossref","first-page":"629","DOI":"10.1056\/NEJMoa0910501","article-title":"Bone marrow transplantation for recessive dystrophic epidermolysis bullosa","volume":"363","author":"Wagner","year":"2010","journal-title":"N\u00a0Engl J Med"},{"key":"10.1016\/j.jid.2018.11.036_bib75","doi-asserted-by":"crossref","first-page":"16014","DOI":"10.1038\/npjregenmed.2016.14","article-title":"CRISPR\/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa","volume":"1","author":"Webber","year":"2016","journal-title":"NPJ Regen Med"},{"issue":"264","key":"10.1016\/j.jid.2018.11.036_bib76","doi-asserted-by":"crossref","first-page":"264ra165","DOI":"10.1126\/scitranslmed.3010083","article-title":"Genetically corrected iPSCs as cell therapy for recessive dystrophic epidermolysis bullosa","volume":"6","author":"Wenzel","year":"2014","journal-title":"Sci Transl Med"},{"key":"10.1016\/j.jid.2018.11.036_bib77","doi-asserted-by":"crossref","first-page":"1138","DOI":"10.1038\/jid.2013.475","article-title":"De novo anti-type VII collagen antibodies in patients with recessive dystrophic epidermolysis bullosa","volume":"134","author":"Woodley","year":"2014","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2018.11.036_bib78","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1038\/nm1063","article-title":"Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa","volume":"10","author":"Woodley","year":"2004","journal-title":"Nat Med"},{"key":"10.1016\/j.jid.2018.11.036_bib79","doi-asserted-by":"crossref","first-page":"318","DOI":"10.1016\/j.ymthe.2004.05.016","article-title":"Intradermal injection of lentiviral vectors corrects regenerated human dystrophic epidermolysis bullosa skin tissue in\u00a0vivo","volume":"10","author":"Woodley","year":"2004","journal-title":"Mol Ther"}],"container-title":["Journal of Investigative Dermatology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0022202X19301319?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0022202X19301319?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,9,19]],"date-time":"2025-09-19T17:14:52Z","timestamp":1758302092000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0022202X19301319"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,6]]},"references-count":79,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2019,6]]}},"alternative-id":["S0022202X19301319"],"URL":"https:\/\/doi.org\/10.1016\/j.jid.2018.11.036","relation":{},"ISSN":["0022-202X"],"issn-type":[{"value":"0022-202X","type":"print"}],"subject":[],"published":{"date-parts":[[2019,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Gene Therapy for Epidermolysis Bullosa","name":"articletitle","label":"Article Title"},{"value":"Journal of Investigative Dermatology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jid.2018.11.036","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2019 The Authors. Published by Elsevier, Inc. on behalf of the Society for Investigative Dermatology.","name":"copyright","label":"Copyright"}]}}