{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,5]],"date-time":"2026-11-05T07:07:00Z","timestamp":1793862420313,"version":"4.6.1"},"reference-count":123,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2015,6,1]],"date-time":"2015-06-01T00:00:00Z","timestamp":1433116800000},"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":494,"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":[[2015,6]]},"DOI":"10.1038\/mt.2015.47","type":"journal-article","created":{"date-parts":[[2015,3,27]],"date-time":"2015-03-27T13:11:40Z","timestamp":1427461900000},"page":"987-992","source":"Crossref","is-referenced-by-count":12,"title":["From Marrow to Matrix: Novel Gene and Cell Therapies for Epidermolysis Bullosa"],"prefix":"10.1016","volume":"23","author":[{"given":"Beau R","family":"Webber","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jakub","family":"Tolar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1038\/mt.2015.47_bib1","doi-asserted-by":"crossref","first-page":"707","DOI":"10.1046\/j.1365-2230.2002.01121.x","article-title":"Quality of life in epidermolysis bullosa","volume":"27","author":"Horn","year":"2002","journal-title":"Clin Exp Dermatol"},{"key":"10.1038\/mt.2015.47_bib2","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1016\/S1357-4310(97)01112-X","article-title":"Epidermolysis bullosa: a spectrum of clinical phenotypes explained by molecular heterogeneity","volume":"3","author":"Uitto","year":"1997","journal-title":"Mol Med Today"},{"key":"10.1038\/mt.2015.47_bib3","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1016\/j.ajo.2004.03.034","article-title":"Eye involvement in inherited epidermolysis bullosa: experience of the National Epidermolysis Bullosa Registry","volume":"138","author":"Fine","year":"2004","journal-title":"Am J Ophthalmol"},{"key":"10.1038\/mt.2015.47_bib4","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":"10.1038\/mt.2015.47_bib5","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/j.det.2009.10.023","article-title":"Understanding the pathogenesis of recessive dystrophic epidermolysis bullosa squamous cell carcinoma","volume":"28","author":"South","year":"2010","journal-title":"Dermatol Clin"},{"key":"10.1038\/mt.2015.47_bib6","doi-asserted-by":"crossref","first-page":"2438","DOI":"10.1038\/sj.jid.5700878","article-title":"Patients with recessive dystrophic epidermolysis bullosa develop squamous-cell carcinoma regardless of type VII collagen expression","volume":"127","author":"Pourreyron","year":"2007","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2015.47_bib7","doi-asserted-by":"crossref","first-page":"1032","DOI":"10.1172\/JCI115916","article-title":"Genetic linkage of recessive dystrophic epidermolysis bullosa to the type VII collagen gene","volume":"90","author":"Hovnanian","year":"1992","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2015.47_bib8","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":"10.1038\/mt.2015.47_bib9","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 Cell Biol"},{"key":"10.1038\/mt.2015.47_bib10","doi-asserted-by":"crossref","first-page":"449","DOI":"10.1016\/j.bbrc.2012.11.119","article-title":"The vWFA2 domain of type VII collagen is responsible for collagen binding","volume":"430","author":"Wegener","year":"2013","journal-title":"Biochem Biophys Res Commun"},{"key":"10.1038\/mt.2015.47_bib11","doi-asserted-by":"crossref","first-page":"1577","DOI":"10.1083\/jcb.103.4.1577","article-title":"Type VII collagen is a major structural component of anchoring fibrils","volume":"103","author":"Sakai","year":"1986","journal-title":"J Cell Biol"},{"key":"10.1038\/mt.2015.47_bib12","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1016\/j.matbio.2013.07.007","article-title":"Disorders of the cutaneous basement membrane zone\u2013the paradigm of epidermolysis bullosa","volume":"33","author":"Bruckner-Tuderman","year":"2014","journal-title":"Matrix Biol"},{"key":"10.1038\/mt.2015.47_bib13","doi-asserted-by":"crossref","first-page":"3498","DOI":"10.1172\/JCI68127","article-title":"Collagen VII plays a dual role in wound healing","volume":"123","author":"Nystr\u00f6m","year":"2013","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2015.47_bib14","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":"10.1038\/mt.2015.47_bib15","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":"10.1038\/mt.2015.47_bib16","doi-asserted-by":"crossref","first-page":"1605","DOI":"10.1038\/mt.2009.144","article-title":"Mechanisms of fibroblast cell therapy for dystrophic epidermolysis bullosa: high stability of collagen VII favors long-term skin integrity","volume":"17","author":"Kern","year":"2009","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2015.47_bib17","doi-asserted-by":"crossref","first-page":"898","DOI":"10.1016\/j.jaad.2013.08.014","article-title":"A phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa","volume":"69","author":"Venugopal","year":"2013","journal-title":"J Am Acad Dermatol"},{"key":"10.1038\/mt.2015.47_bib18","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":"10.1038\/mt.2015.47_bib19","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":"10.1038\/mt.2015.47_bib20","doi-asserted-by":"crossref","first-page":"491","DOI":"10.1056\/NEJM195709122571102","article-title":"Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy","volume":"257","author":"Thomas","year":"1957","journal-title":"N Engl J Med"},{"key":"10.1038\/mt.2015.47_bib21","doi-asserted-by":"crossref","first-page":"1366","DOI":"10.1016\/S0140-6736(68)92673-1","article-title":"Immunological reconstitution of sex-linked lymphopenic immunological deficiency","volume":"2","author":"Gatti","year":"1968","journal-title":"Lancet"},{"key":"10.1038\/mt.2015.47_bib22","doi-asserted-by":"crossref","first-page":"369","DOI":"10.1016\/S0092-8674(01)00328-2","article-title":"Multi-organ, multi-lineage engraftment by a single bone marrow-derived stem cell","volume":"105","author":"Krause","year":"2001","journal-title":"Cell"},{"key":"10.1038\/mt.2015.47_bib23","doi-asserted-by":"crossref","first-page":"738","DOI":"10.1056\/NEJMoa3461002","article-title":"Hepatocytes and epithelial cells of donor origin in recipients of peripheral-blood stem cells","volume":"346","author":"K\u00f6rbling","year":"2002","journal-title":"N Engl J Med"},{"key":"10.1038\/mt.2015.47_bib24","doi-asserted-by":"crossref","first-page":"2663","DOI":"10.1182\/blood-2006-07-033902","article-title":"Donor-derived cells and human graft-versus-host disease of the skin","volume":"109","author":"Murata","year":"2007","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib25","doi-asserted-by":"crossref","first-page":"1658","DOI":"10.1016\/j.bbmt.2010.07.012","article-title":"Nasal epithelial cells of donor origin after allogeneic hematopoietic cell transplantation are generated at a faster rate in the first 3 months compared with later posttransplantation","volume":"16","author":"Khan","year":"2010","journal-title":"Biol Blood Marrow Transplant"},{"key":"10.1038\/mt.2015.47_bib26","doi-asserted-by":"crossref","first-page":"170","DOI":"10.1038\/nrc3217","article-title":"Epithelial stem cells, wound healing and cancer","volume":"12","author":"Arwert","year":"2012","journal-title":"Nat Rev Cancer"},{"key":"10.1038\/mt.2015.47_bib27","doi-asserted-by":"crossref","first-page":"905","DOI":"10.1002\/stem.420","article-title":"Concise review: bone marrow-derived stem\/progenitor cells in cutaneous repair and regeneration","volume":"28","author":"Wu","year":"2010","journal-title":"Stem Cells"},{"key":"10.1038\/mt.2015.47_bib28","doi-asserted-by":"crossref","first-page":"803","DOI":"10.2353\/ajpath.2008.070977","article-title":"Bone marrow cell transfer into fetal circulation can ameliorate genetic skin diseases by providing fibroblasts to the skin and inducing immune tolerance","volume":"173","author":"Chino","year":"2008","journal-title":"Am J Pathol"},{"key":"10.1038\/mt.2015.47_bib29","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":"10.1038\/mt.2015.47_bib30","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1007\/BF03403533","article-title":"Circulating fibrocytes define a new leukocyte subpopulation that mediates tissue repair","volume":"1","author":"Bucala","year":"1994","journal-title":"Mol Med"},{"key":"10.1038\/mt.2015.47_bib31","doi-asserted-by":"crossref","first-page":"6307","DOI":"10.1073\/pnas.94.12.6307","article-title":"The peripheral blood fibrocyte is a potent antigen-presenting cell capable of priming naive T cells in situ","volume":"94","author":"Chesney","year":"1997","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2015.47_bib32","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1038\/nri2990","article-title":"Fibrocytes: emerging effector cells in chronic inflammation","volume":"11","author":"Reilkoff","year":"2011","journal-title":"Nat Rev Immunol"},{"key":"10.1038\/mt.2015.47_bib33","doi-asserted-by":"crossref","first-page":"419","DOI":"10.4049\/jimmunol.160.1.419","article-title":"Regulated production of type I collagen and inflammatory cytokines by peripheral blood fibrocytes","volume":"160","author":"Chesney","year":"1998","journal-title":"J Immunol"},{"key":"10.1038\/mt.2015.47_bib34","doi-asserted-by":"crossref","first-page":"2215","DOI":"10.1096\/fj.01-0049com","article-title":"Fibrocytes induce an angiogenic phenotype in cultured endothelial cells and promote angiogenesis in vivo","volume":"15","author":"Hartlapp","year":"2001","journal-title":"FASEB J"},{"key":"10.1038\/mt.2015.47_bib35","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1111\/j.1524-475X.2006.00192.x","article-title":"Fibrocytes from burn patients regulate the activities of fibroblasts","volume":"15","author":"Wang","year":"2007","journal-title":"Wound Repair Regen"},{"key":"10.1038\/mt.2015.47_bib36","doi-asserted-by":"crossref","first-page":"1347","DOI":"10.1002\/stem.1648","article-title":"Tracking the elusive fibrocyte: identification and characterization of collagen-producing hematopoietic lineage cells during murine wound healing","volume":"32","author":"Suga","year":"2014","journal-title":"Stem Cells"},{"key":"10.1038\/mt.2015.47_bib37","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1002\/jcp.10260","article-title":"Participation of bone marrow derived cells in cutaneous wound healing","volume":"196","author":"Badiavas","year":"2003","journal-title":"J Cell Physiol"},{"key":"10.1038\/mt.2015.47_bib38","doi-asserted-by":"crossref","first-page":"1767","DOI":"10.1016\/S0002-9440(10)63431-1","article-title":"Bone marrow-derived cells contribute to epithelial engraftment during wound healing","volume":"165","author":"Borue","year":"2004","journal-title":"Am J Pathol"},{"key":"10.1038\/mt.2015.47_bib39","doi-asserted-by":"crossref","first-page":"14345","DOI":"10.1073\/pnas.1000044107","article-title":"Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice","volume":"107","author":"Fujita","year":"2010","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2015.47_bib40","doi-asserted-by":"crossref","first-page":"2256","DOI":"10.1126\/science.1074807","article-title":"Little evidence for developmental plasticity of adult hematopoietic stem cells","volume":"297","author":"Wagers","year":"2002","journal-title":"Science"},{"key":"10.1038\/mt.2015.47_bib41","doi-asserted-by":"crossref","first-page":"2648","DOI":"10.1634\/stemcells.2007-0226","article-title":"Mesenchymal stem cells enhance wound healing through differentiation and angiogenesis","volume":"25","author":"Wu","year":"2007","journal-title":"Stem Cells"},{"key":"10.1038\/mt.2015.47_bib42","doi-asserted-by":"crossref","first-page":"110","DOI":"10.1089\/ten.2005.11.110","article-title":"Engrafted bone marrow-derived flk-(1+) mesenchymal stem cells regenerate skin tissue","volume":"11","author":"Deng","year":"2005","journal-title":"Tissue Eng"},{"key":"10.1038\/mt.2015.47_bib43","doi-asserted-by":"crossref","first-page":"154","DOI":"10.1038\/ijos.2014.23","article-title":"Bone marrow-derived mesenchymal stem cells migrate to healthy and damaged salivary glands following stem cell infusion","volume":"6","author":"Schwarz","year":"2014","journal-title":"Int J Oral Sci"},{"key":"10.1038\/mt.2015.47_bib44","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":"10.1038\/mt.2015.47_bib45","doi-asserted-by":"crossref","first-page":"6609","DOI":"10.1073\/pnas.1016753108","article-title":"PDGFRalpha-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":"10.1038\/mt.2015.47_bib46","doi-asserted-by":"crossref","first-page":"1996","DOI":"10.4049\/jimmunol.1400914","article-title":"Transplanted bone marrow-derived circulating PDGFR\u03b1+ cells restore type VII collagen in recessive dystrophic epidermolysis bullosa mouse skin graft","volume":"194","author":"Iinuma","year":"2015","journal-title":"J Immunol"},{"key":"10.1038\/mt.2015.47_bib47","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1016\/j.cell.2006.07.024","article-title":"Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors","volume":"126","author":"Takahashi","year":"2006","journal-title":"Cell"},{"key":"10.1038\/mt.2015.47_bib48","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1016\/j.cell.2007.11.019","article-title":"Induction of pluripotent stem cells from adult human fibroblasts by defined factors","volume":"131","author":"Takahashi","year":"2007","journal-title":"Cell"},{"key":"10.1038\/mt.2015.47_bib49","doi-asserted-by":"crossref","first-page":"391","DOI":"10.1038\/nature10424","article-title":"Targeted gene correction of \u03b11-antitrypsin deficiency in induced pluripotent stem cells","volume":"478","author":"Yusa","year":"2011","journal-title":"Nature"},{"key":"10.1038\/mt.2015.47_bib50","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1186\/scrt89","article-title":"Successful disease-specific induced pluripotent stem cell generation from patients with kidney transplantation","volume":"2","author":"Thatava","year":"2011","journal-title":"Stem Cell Res Ther"},{"key":"10.1038\/mt.2015.47_bib51","doi-asserted-by":"crossref","first-page":"1920","DOI":"10.1126\/science.1152092","article-title":"Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin","volume":"318","author":"Hanna","year":"2007","journal-title":"Science"},{"key":"10.1038\/mt.2015.47_bib52","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":"10.1038\/mt.2015.47_bib53","doi-asserted-by":"crossref","first-page":"562","DOI":"10.1038\/jid.2012.278","article-title":"Keratinocytes from induced pluripotent stem cells in junctional epidermolysis bullosa","volume":"133","author":"Tolar","year":"2013","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2015.47_bib54","doi-asserted-by":"crossref","first-page":"2407","DOI":"10.1038\/jid.2010.163","article-title":"Revertant mosaicism due to a second-site mutation in COL7A1 in a patient with recessive dystrophic epidermolysis bullosa","volume":"130","author":"Pasmooij","year":"2010","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2015.47_bib55","doi-asserted-by":"crossref","first-page":"1937","DOI":"10.1038\/jid.2010.64","article-title":"Revertant mosaicism in recessive dystrophic epidermolysis bullosa","volume":"130","author":"Almaani","year":"2010","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2015.47_bib56","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":"Gosty\u0144ski","year":"2014","journal-title":"J Am Acad Dermatol"},{"key":"10.1038\/mt.2015.47_bib57","doi-asserted-by":"crossref","first-page":"571","DOI":"10.1038\/jid.2013.308","article-title":"Long-term survival of type XVII collagen revertant cells in an animal model of revertant cell therapy","volume":"134","author":"Gosty\u0144ski","year":"2014","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2015.47_bib58","first-page":"167","article-title":"Revertant mosaicism in heritable skin diseases: mechanisms of natural gene therapy","volume":"14","author":"Pasmooij","year":"2012","journal-title":"Discov Med"},{"key":"10.1038\/mt.2015.47_bib59","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":"10.1038\/mt.2015.47_bib60","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":"10.1038\/mt.2015.47_bib61","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 USA"},{"key":"10.1038\/mt.2015.47_bib62","doi-asserted-by":"crossref","first-page":"e77673","DOI":"10.1371\/journal.pone.0077673","article-title":"Generation of 3D skin equivalents fully reconstituted from human induced pluripotent stem cells (iPSCs)","volume":"8","author":"Itoh","year":"2013","journal-title":"PLoS One"},{"issue":"3 Pt 2","key":"10.1038\/mt.2015.47_bib63","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1038\/jid.2011.389","article-title":"Novel molecular therapies for heritable skin disorders","volume":"132","author":"Uitto","year":"2012","journal-title":"J Invest Dermatol"},{"key":"10.1038\/mt.2015.47_bib64","first-page":"1","article-title":"Differentiation of human induced pluripotent stem cells into a keratinocyte lineage","volume":"1195","author":"Kogut","year":"2014","journal-title":"Methods Mol Biol"},{"key":"10.1038\/mt.2015.47_bib65","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":"10.1038\/mt.2015.47_bib66","doi-asserted-by":"crossref","first-page":"1385","DOI":"10.1126\/science.1184733","article-title":"Lgr6 marks stem cells in the hair follicle that generate all cell lineages of the skin","volume":"327","author":"Snippert","year":"2010","journal-title":"Science"},{"key":"10.1038\/mt.2015.47_bib67","doi-asserted-by":"crossref","first-page":"3071","DOI":"10.1038\/ncomms4071","article-title":"Generation of folliculogenic human epithelial stem cells from induced pluripotent stem cells","volume":"5","author":"Yang","year":"2014","journal-title":"Nat Commun"},{"key":"10.1038\/mt.2015.47_bib68","doi-asserted-by":"crossref","first-page":"10716","DOI":"10.1073\/pnas.191362598","article-title":"Hematopoietic colony-forming cells derived from human embryonic stem cells","volume":"98","author":"Kaufman","year":"2001","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2015.47_bib69","doi-asserted-by":"crossref","first-page":"994","DOI":"10.1016\/S0301-472X(03)00261-3","article-title":"Hematopoiesis from embryonic stem cells: lessons from and for ontogeny","volume":"31","author":"Kyba","year":"2003","journal-title":"Exp Hematol"},{"key":"10.1038\/mt.2015.47_bib70","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1242\/dev.118.4.1343","article-title":"ES cells have only a limited lymphopoietic potential after adoptive transfer into mouse recipients","volume":"118","author":"M\u00fcller","year":"1993","journal-title":"Development"},{"key":"10.1038\/mt.2015.47_bib71","doi-asserted-by":"crossref","first-page":"860","DOI":"10.1182\/blood-2004-11-4522","article-title":"Hematopoietic differentiation of human embryonic stem cells progresses through sequential hematoendothelial, primitive, and definitive stages resembling human yolk sac development","volume":"106","author":"Zambidis","year":"2005","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib72","doi-asserted-by":"crossref","first-page":"4035","DOI":"10.1182\/blood-2013-07-474825","article-title":"Hematopoietic specification from human pluripotent stem cells: current advances and challenges toward de novo generation of hematopoietic stem cells","volume":"122","author":"Slukvin","year":"2013","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib73","doi-asserted-by":"crossref","first-page":"1255","DOI":"10.1182\/blood-2012-06-434407","article-title":"In vivo generation of transplantable human hematopoietic cells from induced pluripotent stem cells","volume":"121","author":"Amabile","year":"2013","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib74","doi-asserted-by":"crossref","first-page":"1424","DOI":"10.1038\/mt.2013.71","article-title":"Generation of engraftable hematopoietic stem cells from induced pluripotent stem cells by way of teratoma formation","volume":"21","author":"Suzuki","year":"2013","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2015.47_bib75","doi-asserted-by":"crossref","first-page":"1345","DOI":"10.1126\/science.1191536","article-title":"Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells","volume":"329","author":"Boitano","year":"2010","journal-title":"Science"},{"key":"10.1038\/mt.2015.47_bib76","doi-asserted-by":"crossref","first-page":"1722","DOI":"10.1016\/j.celrep.2012.11.003","article-title":"T lymphocyte potential marks the emergence of definitive hematopoietic progenitors in human pluripotent stem cell differentiation cultures","volume":"2","author":"Kennedy","year":"2012","journal-title":"Cell Rep"},{"key":"10.1038\/mt.2015.47_bib77","doi-asserted-by":"crossref","first-page":"554","DOI":"10.1038\/nbt.2915","article-title":"Wnt signaling controls the specification of definitive and primitive hematopoiesis from human pluripotent stem cells","volume":"32","author":"Sturgeon","year":"2014","journal-title":"Nat Biotechnol"},{"key":"10.1038\/mt.2015.47_bib78","doi-asserted-by":"crossref","first-page":"1509","DOI":"10.1126\/science.1256337","article-title":"Cord blood expansion. Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal","volume":"345","author":"Fares","year":"2014","journal-title":"Science"},{"key":"10.1038\/mt.2015.47_bib79","doi-asserted-by":"crossref","first-page":"e35","DOI":"10.1182\/blood-2012-05-433797","article-title":"Efficient generation, purification, and expansion of CD34(+) hematopoietic progenitor cells from nonhuman primate-induced pluripotent stem cells","volume":"120","author":"Gori","year":"2012","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib80","doi-asserted-by":"crossref","first-page":"312","DOI":"10.1038\/nature13547","article-title":"Reprogramming human endothelial cells to haematopoietic cells requires vascular induction","volume":"511","author":"Sandler","year":"2014","journal-title":"Nature"},{"key":"10.1038\/mt.2015.47_bib81","doi-asserted-by":"crossref","first-page":"549","DOI":"10.1016\/j.cell.2014.04.006","article-title":"Reprogramming committed murine blood cells to induced hematopoietic stem cells with defined factors","volume":"157","author":"Riddell","year":"2014","journal-title":"Cell"},{"key":"10.1038\/mt.2015.47_bib82","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1016\/j.stem.2013.05.024","article-title":"Induction of a hemogenic program in mouse fibroblasts","volume":"13","author":"Pereira","year":"2013","journal-title":"Cell Stem Cell"},{"key":"10.1038\/mt.2015.47_bib83","doi-asserted-by":"crossref","first-page":"350","DOI":"10.1016\/j.exphem.2007.10.007","article-title":"Derivation and immunological characterization of mesenchymal stromal cells from human embryonic stem cells","volume":"36","author":"Trivedi","year":"2008","journal-title":"Exp Hematol"},{"key":"10.1038\/mt.2015.47_bib84","doi-asserted-by":"crossref","first-page":"1517","DOI":"10.1089\/ten.tea.2010.0460","article-title":"Characterization and in vivo testing of mesenchymal stem cells derived from human embryonic stem cells","volume":"17","author":"Gruenloh","year":"2011","journal-title":"Tissue Eng Part A"},{"key":"10.1038\/mt.2015.47_bib85","doi-asserted-by":"crossref","first-page":"530","DOI":"10.1073\/pnas.1423008112","article-title":"MSCs derived from iPSCs with a modified protocol are tumor-tropic but have much less potential to promote tumors than bone marrow MSCs","volume":"112","author":"Zhao","year":"2015","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2015.47_bib86","doi-asserted-by":"crossref","first-page":"4894","DOI":"10.1182\/blood.V87.11.4894.bloodjournal87114894","article-title":"Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome","volume":"87","author":"Peters","year":"1996","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib87","first-page":"43","article-title":"Unrelated donor bone marrow transplantation for Fanconi anemia","volume":"17","author":"Davies","year":"1996","journal-title":"Bone Marrow Transplant"},{"key":"10.1038\/mt.2015.47_bib88","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1038\/bmt.2010.65","article-title":"Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita","volume":"46","author":"Dietz","year":"2011","journal-title":"Bone Marrow Transplant"},{"key":"10.1038\/mt.2015.47_bib89","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1038\/sj.bmt.1705533","article-title":"Engraftment and survival following hematopoietic stem cell transplantation for osteopetrosis using a reduced intensity conditioning regimen","volume":"38","author":"Tolar","year":"2006","journal-title":"Bone Marrow Transplant"},{"key":"10.1038\/mt.2015.47_bib90","doi-asserted-by":"crossref","first-page":"2262","DOI":"10.1182\/blood-2013-05-502658","article-title":"Long-term health-related quality of life evaluated more than 20 years after hematopoietic stem cell transplantation for thalassemia","volume":"122","author":"La Nasa","year":"2013","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib91","doi-asserted-by":"crossref","first-page":"4279","DOI":"10.1182\/blood-2013-01-479733","article-title":"Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the European Group for Blood and Marrow Transplantation experience","volume":"122","author":"Peffault de Latour","year":"2013","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib92","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1111\/j.1365-2141.2010.08078.x","article-title":"Haematopoeitic cell transplantation for Fanconi anaemia - when and how?","volume":"149","author":"MacMillan","year":"2010","journal-title":"Br J Haematol"},{"key":"10.1038\/mt.2015.47_bib93","doi-asserted-by":"crossref","first-page":"3981","DOI":"10.1182\/blood-2012-09-455238","article-title":"Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning","volume":"121","author":"Boelens","year":"2013","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib94","doi-asserted-by":"crossref","first-page":"1509","DOI":"10.1038\/mt.2010.91","article-title":"SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa","volume":"18","author":"Titeux","year":"2010","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2015.47_bib95","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.2015.47_bib96","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":"10.1038\/mt.2015.47_bib97","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":"10.1038\/mt.2015.47_bib98","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":"10.1038\/mt.2015.47_bib99","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1126\/science.1171242","article-title":"Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy","volume":"326","author":"Cartier","year":"2009","journal-title":"Science"},{"key":"10.1038\/mt.2015.47_bib100","doi-asserted-by":"crossref","first-page":"1233151","DOI":"10.1126\/science.1233151","article-title":"Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome","volume":"341","author":"Aiuti","year":"2013","journal-title":"Science"},{"key":"10.1038\/mt.2015.47_bib101","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":"10.1038\/mt.2015.47_bib102","doi-asserted-by":"crossref","first-page":"1407","DOI":"10.1056\/NEJMoa1404588","article-title":"A modified \u03b3-retrovirus vector for X-linked severe combined immunodeficiency","volume":"371","author":"Hacein-Bey-Abina","year":"2014","journal-title":"N Engl J Med"},{"key":"10.1038\/mt.2015.47_bib103","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":"10.1038\/mt.2015.47_bib104","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1038\/msb.2013.17","article-title":"Global remodelling of cellular microenvironment due to loss of collagen VII","volume":"9","author":"K\u00fcttner","year":"2013","journal-title":"Mol Syst Biol"},{"key":"10.1038\/mt.2015.47_bib105","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":"10.1038\/mt.2015.47_bib106","doi-asserted-by":"crossref","first-page":"763","DOI":"10.1126\/science.1078395","article-title":"Chimeric nucleases stimulate gene targeting in human cells","volume":"300","author":"Porteus","year":"2003","journal-title":"Science"},{"key":"10.1038\/mt.2015.47_bib107","doi-asserted-by":"crossref","first-page":"646","DOI":"10.1038\/nature03556","article-title":"Highly efficient endogenous human gene correction using designed zinc-finger nucleases","volume":"435","author":"Urnov","year":"2005","journal-title":"Nature"},{"key":"10.1038\/mt.2015.47_bib108","doi-asserted-by":"crossref","first-page":"3906","DOI":"10.1182\/blood-2012-03-420703","article-title":"Zinc-finger nuclease-mediated correction of \u03b1-thalassemia in iPS cells","volume":"120","author":"Chang","year":"2012","journal-title":"Blood"},{"key":"10.1038\/mt.2015.47_bib109","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":"10.1038\/mt.2015.47_bib110","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"},{"key":"10.1038\/mt.2015.47_bib111","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":"10.1038\/mt.2015.47_bib112","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":"10.1038\/mt.2015.47_bib113","doi-asserted-by":"crossref","first-page":"910","DOI":"10.1016\/j.cell.2013.04.025","article-title":"One-step generation of mice carrying mutations in multiple genes by CRISPR\/Cas-mediated genome engineering","volume":"153","author":"Wang","year":"2013","journal-title":"Cell"},{"key":"10.1038\/mt.2015.47_bib114","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":"10.1038\/mt.2015.47_bib115","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":"10.1038\/mt.2015.47_bib116","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1097\/TP.0000000000000204","article-title":"Simultaneous transplantation of hematopoietic stem cells and a vascularized composite allograft leads to tolerance","volume":"98","author":"Mathes","year":"2014","journal-title":"Transplantation"},{"key":"10.1038\/mt.2015.47_bib117","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1002\/path.2441","article-title":"Epithelial stem cells in corneal regeneration and epidermal gene therapy","volume":"217","author":"Pellegrini","year":"2009","journal-title":"J Pathol"},{"key":"10.1038\/mt.2015.47_bib118","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1038\/sj.cr.7310137","article-title":"Niche regulation of corneal epithelial stem cells at the limbus","volume":"17","author":"Li","year":"2007","journal-title":"Cell Res"},{"key":"10.1038\/mt.2015.47_bib119","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":"10.1038\/mt.2015.47_bib120","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":"10.1038\/mt.2015.47_bib121","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":"10.1038\/mt.2015.47_bib122","doi-asserted-by":"crossref","first-page":"1741","DOI":"10.1038\/mt.2014.140","article-title":"Aminoglycosides restore full-length type VII collagen by overcoming premature termination codons: therapeutic implications for dystrophic epidermolysis bullosa","volume":"22","author":"Cogan","year":"2014","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2015.47_bib123","doi-asserted-by":"crossref","first-page":"2458","DOI":"10.1038\/jid.2014.162","article-title":"Pharmacological mobilization of endogenous stem cells significantly promotes skin regeneration after full-thickness excision: the synergistic activity of AMD3100 and tacrolimus","volume":"134","author":"Lin","year":"2014","journal-title":"J Invest Dermatol"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616301277?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616301277?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2023,8,9]],"date-time":"2023-08-09T01:03:47Z","timestamp":1691543027000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616301277"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,6]]},"references-count":123,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2015,6]]}},"alternative-id":["S1525001616301277"],"URL":"https:\/\/doi.org\/10.1038\/mt.2015.47","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2015,6]]}}}