{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,9]],"date-time":"2026-07-09T23:20:20Z","timestamp":1783639220361,"version":"3.55.0"},"reference-count":88,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2016,2,1]],"date-time":"2016-02-01T00:00:00Z","timestamp":1454284800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,2,1]],"date-time":"2016-02-01T00:00:00Z","timestamp":1454284800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2017,1,14]],"date-time":"2017-01-14T00:00:00Z","timestamp":1484352000000},"content-version":"am","delay-in-days":348,"URL":"http:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R01 AR063070"],"award-info":[{"award-number":["R01 AR063070"]}],"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":["R01AR059947"],"award-info":[{"award-number":["R01AR059947"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000005","name":"U.S. Department of Defense","doi-asserted-by":"publisher","award":["W81XWH-12-1-0609"],"award-info":[{"award-number":["W81XWH-12-1-0609"]}],"id":[{"id":"10.13039\/100000005","id-type":"DOI","asserted-by":"publisher"}]},{"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"}]},{"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\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["UL1TR000114"],"award-info":[{"award-number":["UL1TR000114"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","translationalres.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Translational Research"],"published-print":{"date-parts":[[2016,2]]},"DOI":"10.1016\/j.trsl.2015.05.008","type":"journal-article","created":{"date-parts":[[2015,5,26]],"date-time":"2015-05-26T22:17:01Z","timestamp":1432678621000},"page":"50-58","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":18,"special_numbering":"C","title":["Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa"],"prefix":"10.1016","volume":"168","author":[{"given":"Christopher","family":"Perdoni","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark J.","family":"Osborn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jakub","family":"Tolar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.trsl.2015.05.008_bib1","doi-asserted-by":"crossref","first-page":"1103","DOI":"10.1016\/j.jaad.2014.01.903","article-title":"Inherited epidermolysis bullosa: updated recommendations on diagnosis and classification","volume":"70","author":"Fine","year":"2014","journal-title":"J Am Acad Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib2","doi-asserted-by":"crossref","first-page":"366","DOI":"10.1111\/1523-1747.ep12471830","article-title":"Structural variations in anchoring fibrils in dystrophic epidermolysis bullosa: correlation with type VII collagen expression","volume":"100","author":"McGrath","year":"1993","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib3","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1056\/NEJMcibr1414709","article-title":"A biologic Velcro patch","volume":"372","author":"Tolar","year":"2015","journal-title":"N Engl J Med"},{"key":"10.1016\/j.trsl.2015.05.008_bib4","doi-asserted-by":"crossref","first-page":"387","DOI":"10.1016\/j.jaad.2009.03.053","article-title":"Extracutaneous manifestations and complications of inherited epidermolysis bullosa: part II. Other organs","volume":"61","author":"Fine","year":"2009","journal-title":"J Am Acad Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib5","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/j.det.2009.10.020","article-title":"Dystrophic epidermolysis bullosa: pathogenesis and clinical features","volume":"28","author":"Bruckner-Tuderman","year":"2010","journal-title":"Dermatol Clin"},{"key":"10.1016\/j.trsl.2015.05.008_bib6","doi-asserted-by":"crossref","first-page":"483","DOI":"10.1046\/j.1523-1747.2001.01279-11.x","article-title":"Epidermolysis bullosa carrier frequencies in the US population","volume":"116","author":"Pfendner","year":"2001","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib7","series-title":"Life with epidermolysis bullosa (EB): etiology, diagnosis, multidisciplinary care and therapy","first-page":"197","article-title":"Premature death in epidermolysis bullosa","author":"Fine","year":"2009"},{"key":"10.1016\/j.trsl.2015.05.008_bib8","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 alpha 1(VII) chain and identification of intragenic polymorphisms","volume":"269","author":"Christiano","year":"1994","journal-title":"J Biol Chem"},{"key":"10.1016\/j.trsl.2015.05.008_bib9","doi-asserted-by":"crossref","first-page":"369","DOI":"10.1358\/dnp.2008.21.7.1255294","article-title":"Proteinase-activated receptor-2 in the skin: receptor expression, activation and function during health and disease","volume":"21","author":"Rattenholl","year":"2008","journal-title":"Drug News Perspect"},{"key":"10.1016\/j.trsl.2015.05.008_bib10","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1111\/j.1600-0625.2008.00723.x","article-title":"Mutation analysis and characterization of COL7A1 mutations in dystrophic epidermolysis bullosa","volume":"17","author":"Dang","year":"2008","journal-title":"Exp Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib11","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1007\/BF00370713","article-title":"Molecular basis for the dystrophic forms of epidermolysis bullosa: mutations in the type VII collagen gene","volume":"287","author":"Uitto","year":"1994","journal-title":"Arch Dermatol Res"},{"key":"10.1016\/j.trsl.2015.05.008_bib12","doi-asserted-by":"crossref","first-page":"19228","DOI":"10.1074\/jbc.273.30.19228","article-title":"Some, but not all, glycine substitution mutations in COL7A1 result in intracellular accumulation of collagen VII, loss of anchoring fibrils, and skin blistering","volume":"273","author":"Hammami-Hauasli","year":"1998","journal-title":"J Biol Chem"},{"key":"10.1016\/j.trsl.2015.05.008_bib13","first-page":"893","article-title":"Two cases of recessive dystrophic epidermolysis bullosa diagnosed as severe generalized","volume":"38","author":"Ohashi","year":"2011","journal-title":"J Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib14","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.trsl.2015.05.008_bib15","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.trsl.2015.05.008_bib16","doi-asserted-by":"crossref","first-page":"1910","DOI":"10.1038\/jid.2013.10","article-title":"Intravenously injected recombinant human type VII collagen homes to skin wounds and restores skin integrity of dystrophic epidermolysis bullosa","volume":"133","author":"Woodley","year":"2013","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib17","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1038\/mt.2008.262","article-title":"Can type VII collagen injections cure dystrophic epidermolysis bullosa?","volume":"17","author":"Bruckner-Tuderman","year":"2009","journal-title":"Mol Ther"},{"key":"10.1016\/j.trsl.2015.05.008_bib18","doi-asserted-by":"crossref","first-page":"2179","DOI":"10.1038\/jid.2008.78","article-title":"Potential of fibroblast cell therapy for recessive dystrophic epidermolysis bullosa","volume":"128","author":"Wong","year":"2008","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib19","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.trsl.2015.05.008_bib20","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.trsl.2015.05.008_bib21","doi-asserted-by":"crossref","first-page":"30","DOI":"10.3109\/14653249.2010.518609","article-title":"Gene expression signatures of mouse bone marrow-derived mesenchymal stem cells in the cutaneous environment and therapeutic implications for blistering skin disorder","volume":"13","author":"Alexeev","year":"2011","journal-title":"Cytotherapy"},{"key":"10.1016\/j.trsl.2015.05.008_bib22","doi-asserted-by":"crossref","first-page":"32","DOI":"10.1038\/icb.2012.64","article-title":"Immunological characteristics of human mesenchymal stem cells and multipotent adult progenitor cells","volume":"91","author":"Jacobs","year":"2013","journal-title":"Immunol Cell Biol"},{"key":"10.1016\/j.trsl.2015.05.008_bib23","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 Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib24","doi-asserted-by":"crossref","first-page":"925","DOI":"10.1007\/s00392-011-0327-y","article-title":"Transplantation of progenitor cells and regeneration enhancement in acute myocardial infarction (TOPCARE-AMI): final 5-year results suggest long-term safety and efficacy","volume":"100","author":"Leistner","year":"2011","journal-title":"Clin Res Cardiol"},{"key":"10.1016\/j.trsl.2015.05.008_bib25","doi-asserted-by":"crossref","first-page":"3300","DOI":"10.1161\/01.CIR.0000147780.30124.CF","article-title":"Stromal cell-derived factor-1alpha plays a critical role in stem cell recruitment to the heart after myocardial infarction but is not sufficient to induce homing in the absence of injury","volume":"110","author":"Abbott","year":"2004","journal-title":"Circulation"},{"key":"10.1016\/j.trsl.2015.05.008_bib26","doi-asserted-by":"crossref","first-page":"1167","DOI":"10.1182\/blood-2008-06-161299","article-title":"Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells","volume":"113","author":"Tolar","year":"2009","journal-title":"Blood"},{"key":"10.1016\/j.trsl.2015.05.008_bib27","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 Engl J Med"},{"key":"10.1016\/j.trsl.2015.05.008_bib28","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1186\/scrt511","article-title":"Preconditioning of mesenchymal stem cells for improved transplantation efficacy in recessive dystrophic epidermolysis bullosa","volume":"5","author":"Perdoni","year":"2014","journal-title":"Stem Cell Res Ther"},{"key":"10.1016\/j.trsl.2015.05.008_bib29","doi-asserted-by":"crossref","first-page":"766","DOI":"10.1038\/sj.jid.5700117","article-title":"Fibroblasts show more potential as target cells than keratinocytes in COL7A1 gene therapy of dystrophic epidermolysis bullosa","volume":"126","author":"Goto","year":"2006","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib30","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 Clin Invest"},{"key":"10.1016\/j.trsl.2015.05.008_bib31","doi-asserted-by":"crossref","first-page":"2289","DOI":"10.1089\/104303400750035834","article-title":"Fibroblasts and dermal gene therapy: a mini review","volume":"11","author":"Krueger","year":"2000","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.trsl.2015.05.008_bib32","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.trsl.2015.05.008_bib33","doi-asserted-by":"crossref","first-page":"1105","DOI":"10.1038\/nm779","article-title":"CDK4 coexpression with Ras generates malignant human epidermal tumorigenesis","volume":"8","author":"Lazarov","year":"2002","journal-title":"Nat Med"},{"key":"10.1016\/j.trsl.2015.05.008_bib34","doi-asserted-by":"crossref","first-page":"1897","DOI":"10.1093\/hmg\/ddg200","article-title":"Genetic correction of canine dystrophic epidermolysis bullosa mediated by retroviral vectors","volume":"12","author":"Baldeschi","year":"2003","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.trsl.2015.05.008_bib35","doi-asserted-by":"crossref","first-page":"1166","DOI":"10.1038\/nm766","article-title":"Stable nonviral genetic correction of inherited human skin disease","volume":"8","author":"Ortiz-Urda","year":"2002","journal-title":"Nat Med"},{"key":"10.1016\/j.trsl.2015.05.008_bib36","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.trsl.2015.05.008_bib37","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.trsl.2015.05.008_bib38","doi-asserted-by":"crossref","first-page":"848","DOI":"10.1038\/jid.2010.346","article-title":"Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa","volume":"131","author":"Tolar","year":"2011","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib39","doi-asserted-by":"crossref","first-page":"8797","DOI":"10.1073\/pnas.1100332108","article-title":"Generation of keratinocytes from normal and recessive dystrophic epidermolysis bullosa-induced pluripotent stem cells","volume":"108","author":"Itoh","year":"2011","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.trsl.2015.05.008_bib40","doi-asserted-by":"crossref","first-page":"264ra164","DOI":"10.1126\/scitranslmed.3009342","article-title":"Induced pluripotent stem cells from human revertant keratinocytes for the treatment of epidermolysis bullosa","volume":"6","author":"Umegaki-Arao","year":"2014","journal-title":"Sci Transl Med"},{"key":"10.1016\/j.trsl.2015.05.008_bib41","doi-asserted-by":"crossref","first-page":"967","DOI":"10.1046\/j.1523-1747.2002.01756.x","article-title":"The majority of keratinocytes incorporate intradermally injected plasmid DNA regardless of size but only a small proportion of cells can express the gene product","volume":"118","author":"Sawamura","year":"2002","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib42","doi-asserted-by":"crossref","first-page":"1655","DOI":"10.1089\/10430340260201743","article-title":"A microinjected COL7A1-PAC vector restores synthesis of intact procollagen VII in a dystrophic epidermolysis bullosa keratinocyte cell line","volume":"13","author":"Mecklenbeck","year":"2002","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.trsl.2015.05.008_bib43","doi-asserted-by":"crossref","first-page":"24429","DOI":"10.1074\/jbc.M003440200","article-title":"Development and characterization of a recombinant truncated type VII collagen \u201cminigene\u201d. Implication for gene therapy of dystrophic epidermolysis bullosa","volume":"275","author":"Chen","year":"2000","journal-title":"J Biol Chem"},{"key":"10.1016\/j.trsl.2015.05.008_bib44","doi-asserted-by":"crossref","first-page":"670","DOI":"10.1038\/ng1041","article-title":"Restoration of type VII collagen expression and function in dystrophic epidermolysis bullosa","volume":"32","author":"Chen","year":"2002","journal-title":"Nat Genet"},{"key":"10.1016\/j.trsl.2015.05.008_bib45","doi-asserted-by":"crossref","first-page":"1021","DOI":"10.1046\/j.1523-1747.2003.12571.x","article-title":"Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone","volume":"121","author":"Woodley","year":"2003","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib46","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"},{"key":"10.1016\/j.trsl.2015.05.008_bib47","doi-asserted-by":"crossref","first-page":"1749","DOI":"10.1126\/science.1083413","article-title":"Transcription start regions in the human genome are favored targets for MLV integration","volume":"300","author":"Wu","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.trsl.2015.05.008_bib48","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.trsl.2015.05.008_bib49","doi-asserted-by":"crossref","first-page":"3132","DOI":"10.1172\/JCI35700","article-title":"Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1","volume":"118","author":"Hacein-Bey-Abina","year":"2008","journal-title":"J Clin Invest"},{"key":"10.1016\/j.trsl.2015.05.008_bib50","first-page":"255","article-title":"Gene therapy of x-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vector","volume":"194","author":"Cartier","year":"2010","journal-title":"Bull Acad Natl Med"},{"key":"10.1016\/j.trsl.2015.05.008_bib51","doi-asserted-by":"crossref","first-page":"1233158","DOI":"10.1126\/science.1233158","article-title":"Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy","volume":"341","author":"Biffi","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.trsl.2015.05.008_bib52","doi-asserted-by":"crossref","first-page":"5193","DOI":"10.1073\/pnas.091484998","article-title":"In\u00a0vivo restoration of laminin 5 beta 3 expression and function in junctional epidermolysis bullosa","volume":"98","author":"Robbins","year":"2001","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"6679 suppl","key":"10.1016\/j.trsl.2015.05.008_bib53","first-page":"25","article-title":"Human gene therapy","volume":"392","author":"Anderson","year":"1998","journal-title":"Nature"},{"key":"10.1016\/j.trsl.2015.05.008_bib54","doi-asserted-by":"crossref","first-page":"761","DOI":"10.1586\/erv.09.29","article-title":"Strategies to overcome host immunity to adenovirus vectors in vaccine development","volume":"8","author":"Thacker","year":"2009","journal-title":"Expert Rev Vaccines"},{"key":"10.1016\/j.trsl.2015.05.008_bib55","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1177\/009885881003600202","article-title":"The death of Jesse Gelsinger: new evidence of the influence of money and prestige in human research","volume":"36","author":"Wilson","year":"2010","journal-title":"Am J Law Med"},{"key":"10.1016\/j.trsl.2015.05.008_bib56","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1038\/jid.2010.249","article-title":"Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa","volume":"131","author":"Murauer","year":"2011","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib57","first-page":"S75","article-title":"Phase I clinical trial of genetically corrected autologous epidermal keratinocytes for recessive dystrophic epidermolysis bullosa","volume":"134","author":"Siprashvili","year":"2014","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib58","doi-asserted-by":"crossref","first-page":"1770","DOI":"10.1182\/blood-2007-01-068759","article-title":"Hot spots of retroviral integration in human CD34+ hematopoietic cells","volume":"110","author":"Cattoglio","year":"2007","journal-title":"Blood"},{"key":"10.1016\/j.trsl.2015.05.008_bib59","first-page":"442","article-title":"Adeno-associated virus vector integration","volume":"11","author":"Deyle","year":"2009","journal-title":"Curr Opin Mol Ther"},{"key":"10.1016\/j.trsl.2015.05.008_bib60","doi-asserted-by":"crossref","first-page":"6386","DOI":"10.1128\/MCB.17.11.6386","article-title":"Chromosomal double-strand breaks induce gene conversion at high frequency in mammalian cells","volume":"17","author":"Taghian","year":"1997","journal-title":"Mol Cell Biol"},{"key":"10.1016\/j.trsl.2015.05.008_bib61","doi-asserted-by":"crossref","first-page":"4070","DOI":"10.1128\/MCB.18.7.4070","article-title":"Analysis of gene targeting and intrachromosomal homologous recombination stimulated by genomic double-strand breaks in mouse embryonic stem cells","volume":"18","author":"Donoho","year":"1998","journal-title":"Mol Cell Biol"},{"key":"10.1016\/j.trsl.2015.05.008_bib62","doi-asserted-by":"crossref","first-page":"294","DOI":"10.1016\/j.molcel.2008.06.016","article-title":"Rapid \u201copen-source\u201d engineering of customized zinc-finger nucleases for highly efficient gene modification","volume":"31","author":"Maeder","year":"2008","journal-title":"Mol Cell"},{"key":"10.1016\/j.trsl.2015.05.008_bib63","doi-asserted-by":"crossref","first-page":"438","DOI":"10.1016\/j.ymthe.2005.08.003","article-title":"Mammalian gene targeting with designed zinc finger nucleases","volume":"13","author":"Porteus","year":"2006","journal-title":"Mol Ther"},{"key":"10.1016\/j.trsl.2015.05.008_bib64","doi-asserted-by":"crossref","first-page":"967","DOI":"10.1038\/nbt1125","article-title":"Gene targeting using zinc finger nucleases","volume":"23","author":"Porteus","year":"2005","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.trsl.2015.05.008_bib65","doi-asserted-by":"crossref","first-page":"757","DOI":"10.1534\/genetics.110.120717","article-title":"Targeting DNA double-strand breaks with TAL effector nucleases","volume":"186","author":"Christian","year":"2010","journal-title":"Genetics"},{"key":"10.1016\/j.trsl.2015.05.008_bib66","doi-asserted-by":"crossref","first-page":"e82","DOI":"10.1093\/nar\/gkr218","article-title":"Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting","volume":"39","author":"Cermak","year":"2011","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.trsl.2015.05.008_bib67","doi-asserted-by":"crossref","first-page":"1501","DOI":"10.1126\/science.1178817","article-title":"A simple cipher governs DNA recognition by TAL effectors","volume":"326","author":"Moscou","year":"2009","journal-title":"Science"},{"key":"10.1016\/j.trsl.2015.05.008_bib68","doi-asserted-by":"crossref","first-page":"1509","DOI":"10.1126\/science.1178811","article-title":"Breaking the code of DNA binding specificity of TAL-type III effectors","volume":"326","author":"Boch","year":"2009","journal-title":"Science"},{"key":"10.1016\/j.trsl.2015.05.008_bib69","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1038\/nbt.1755","article-title":"A TALE nuclease architecture for efficient genome editing","volume":"29","author":"Miller","year":"2011","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.trsl.2015.05.008_bib70","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"Jinek","year":"2012","journal-title":"Science"},{"key":"10.1016\/j.trsl.2015.05.008_bib71","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.trsl.2015.05.008_bib72","doi-asserted-by":"crossref","first-page":"569","DOI":"10.1038\/nature13579","article-title":"Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease","volume":"513","author":"Anders","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.trsl.2015.05.008_bib73","doi-asserted-by":"crossref","first-page":"1491","DOI":"10.1126\/science.1183604","article-title":"Plant science. DNA binding made easy","volume":"326","author":"Voytas","year":"2009","journal-title":"Science"},{"key":"10.1016\/j.trsl.2015.05.008_bib74","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1038\/nmeth.1542","article-title":"Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA)","volume":"8","author":"Sander","year":"2011","journal-title":"Nat Methods"},{"key":"10.1016\/j.trsl.2015.05.008_bib75","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.trsl.2015.05.008_bib76","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1038\/nbt.1948","article-title":"An unbiased genome-wide analysis of zinc-finger nuclease specificity","volume":"29","author":"Gabriel","year":"2011","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.trsl.2015.05.008_bib77","doi-asserted-by":"crossref","first-page":"9584","DOI":"10.1093\/nar\/gkt714","article-title":"CRISPR\/Cas9 systems targeting beta-globin and CCR5 genes have substantial off-target activity","volume":"41","author":"Cradick","year":"2013","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.trsl.2015.05.008_bib78","doi-asserted-by":"crossref","DOI":"10.1128\/mBio.01114-14","article-title":"Efficient gene disruption in diverse strains of Toxoplasma gondii using CRISPR\/CAS9","volume":"5","author":"Shen","year":"2014","journal-title":"MBio"},{"key":"10.1016\/j.trsl.2015.05.008_bib79","doi-asserted-by":"crossref","first-page":"114","DOI":"10.1089\/hum.2014.111","article-title":"Fanconi anemia gene editing by the CRISPR\/Cas9 system","volume":"26","author":"Osborn","year":"2015","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.trsl.2015.05.008_bib80","doi-asserted-by":"crossref","first-page":"9283","DOI":"10.1093\/nar\/gkr597","article-title":"A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity","volume":"39","author":"Mussolino","year":"2011","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.trsl.2015.05.008_bib81","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1038\/nbt.3127","article-title":"Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors","volume":"33","author":"Wang","year":"2015","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.trsl.2015.05.008_bib82","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1038\/nbt.3101","article-title":"Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases","volume":"33","author":"Frock","year":"2015","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.trsl.2015.05.008_bib83","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1038\/nbt.3117","article-title":"GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases","volume":"33","author":"Tsai","year":"2015","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.trsl.2015.05.008_bib84","doi-asserted-by":"crossref","first-page":"3550","DOI":"10.1128\/MCB.23.10.3550-3557.2003","article-title":"Human gene targeting by adeno-associated virus vectors is enhanced by DNA double-strand breaks","volume":"23","author":"Miller","year":"2003","journal-title":"Mol Cell Biol"},{"key":"10.1016\/j.trsl.2015.05.008_bib85","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 Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib86","doi-asserted-by":"crossref","first-page":"2097","DOI":"10.1038\/jid.2014.118","article-title":"Mechanisms of natural gene therapy in dystrophic epidermolysis bullosa","volume":"134","author":"Kiritsi","year":"2014","journal-title":"J Invest Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib87","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1016\/j.jaad.2013.08.052","article-title":"Successful therapeutic transplantation of revertant skin in epidermolysis bullosa","volume":"70","author":"Gostynski","year":"2014","journal-title":"J Am Acad Dermatol"},{"key":"10.1016\/j.trsl.2015.05.008_bib88","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1038\/nature13420","article-title":"Targeted genome editing in human repopulating haematopoietic stem cells","volume":"510","author":"Genovese","year":"2014","journal-title":"Nature"}],"container-title":["Translational Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1931524415001759?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1931524415001759?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,7,9]],"date-time":"2026-07-09T23:05:04Z","timestamp":1783638304000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1931524415001759"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,2]]},"references-count":88,"alternative-id":["S1931524415001759"],"URL":"https:\/\/doi.org\/10.1016\/j.trsl.2015.05.008","relation":{},"ISSN":["1931-5244"],"issn-type":[{"value":"1931-5244","type":"print"}],"subject":[],"published":{"date-parts":[[2016,2]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa","name":"articletitle","label":"Article Title"},{"value":"Translational Research","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.trsl.2015.05.008","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2016 Elsevier Inc. All rights reserved.","name":"copyright","label":"Copyright"}]}}