{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T18:58:50Z","timestamp":1790621930362,"version":"4.1.0"},"reference-count":23,"publisher":"American Society for Clinical Investigation","issue":"19","funder":[{"name":"NIH","award":["R01 AR055914"],"award-info":[{"award-number":["R01 AR055914"]}]},{"name":"EB Research Partnership","award":["n\/a"],"award-info":[{"award-number":["n\/a"]}]},{"DOI":"10.13039\/100002077","name":"EB Medical Research Foundation","doi-asserted-by":"crossref","award":["n\/a"],"award-info":[{"award-number":["n\/a"]}],"id":[{"id":"10.13039\/100002077","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2019,10,3]]},"abstract":"<jats:sec>\n                    <jats:title>BACKGROUND<\/jats:title>\n                    <jats:p>\n                      Recessive dystrophic epidermolysis bullosa (RDEB) patients have mutations in the\n                      <jats:italic>COL7A1<\/jats:italic>\n                      gene and thus lack functional type VII collagen (C7) protein; they have marked skin fragility and blistering. This single-center phase 1\/2a open-label study evaluated the long-term efficacy, safety, and patient-reported outcomes in RDEB patients treated with gene-corrected autologous cell therapy.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>METHODS<\/jats:title>\n                    <jats:p>\n                      Autologous keratinocytes were isolated from participant skin biopsies. Epidermal sheets were prepared from cells transduced with a retrovirus carrying the full-length human\n                      <jats:italic>COL7A1<\/jats:italic>\n                      gene. These gene-corrected autologous epidermal sheets measured 5 \u00d7 7 cm (35 cm\n                      <jats:sup>2<\/jats:sup>\n                      ) and were transplanted onto 6 wound sites in each of 7 adult participants (\n                      <jats:italic>n<\/jats:italic>\n                      = 42 sites total) from 2013 to 2017. Participants were followed for 2 to 5 years.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>RESULTS<\/jats:title>\n                    <jats:p>\n                      No participants experienced any serious related adverse events. Wound healing of 50% or greater by Investigator Global Assessment was present in 95% (36 of 38) of treated wounds versus 0% (0 of 6) of untreated control wounds at 6 months (\n                      <jats:italic>P<\/jats:italic>\n                      &lt; 0.0001). At year 1, 68% (26 of 38) of treated wounds had 50% or greater healing compared with 17% (1 of 6) of control wounds (\n                      <jats:italic>P<\/jats:italic>\n                      = 0.025). At year 2, 71% (27 of 38) of treated wounds had 50% or greater healing compared with 17% (1 of 6) of control wounds (\n                      <jats:italic>P<\/jats:italic>\n                      = 0.019).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>CONCLUSION<\/jats:title>\n                    <jats:p>C7 expression persisted up to 2 years after treatment in 2 participants. Treated wounds with 50% or greater healing demonstrated improvement in patient-reported pain, itch, and wound durability. This study provides additional data to support the clinically meaningful benefit of treating chronic RDEB wounds with ex vivo, C7 gene\u2013corrected autologous cell therapy. This approach was safe and promoted wound healing that was associated with improved patient-reported outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>TRIAL REGISTRATION<\/jats:title>\n                    <jats:p>Clinicaltrials.gov identifier: NCT01263379.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>FUNDING<\/jats:title>\n                    <jats:p>Epidermolysis Bullosa Research Partnership, Epidermolysis Bullosa Medical Research Foundation, NIH R01 AR055914, Office of Research and Development at the Palo Alto Veteran\u2019s Affairs Medical Center, and the Dermatology Foundation.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1172\/jci.insight.130554","type":"journal-article","created":{"date-parts":[[2019,10,2]],"date-time":"2019-10-02T11:00:55Z","timestamp":1570014055000},"source":"Crossref","is-referenced-by-count":108,"title":["Phase 1\/2a clinical trial of gene-corrected autologous cell therapy for recessive dystrophic epidermolysis bullosa"],"prefix":"10.1172","volume":"4","author":[{"given":"Shaundra","family":"Eichstadt","sequence":"first","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Barriga","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anusha","family":"Ponakala","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Teng","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ngon T.","family":"Nguyen","sequence":"additional","affiliation":[{"name":"Abeona Therapeutics, Cleveland, Ohio, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zurab","family":"Siprashvili","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaron","family":"Nazaroff","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily S.","family":"Gorell","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Albert S.","family":"Chiou","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa","family":"Taylor","sequence":"additional","affiliation":[{"name":"Lucile Packard Children\u2019s Hospital, Stanford Children\u2019s Health, Stanford, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Phuong","family":"Khuu","sequence":"additional","affiliation":[{"name":"Lucile Packard Children\u2019s Hospital, Stanford Children\u2019s Health, Stanford, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas R.","family":"Keene","sequence":"additional","affiliation":[{"name":"Shriners Hospital for Children, Portland, Oregon, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4436-2471","authenticated-orcid":true,"given":"Kerri","family":"Rieger","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rohit K.","family":"Khosla","sequence":"additional","affiliation":[{"name":"Stanford University School of Medicine, Department of Dermatology, Redwood City, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Louise K.","family":"Furukawa","sequence":"additional","affiliation":[{"name":"Lucile Packard Children\u2019s Hospital, Stanford Children\u2019s Health, Stanford, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H. 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