{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,24]],"date-time":"2026-08-24T15:40:27Z","timestamp":1787586027888,"version":"build-2736575974"},"reference-count":73,"publisher":"Elsevier BV","issue":"12","license":[{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,12,1]],"date-time":"2025-12-01T00:00:00Z","timestamp":1764547200000},"content-version":"vor","delay-in-days":365,"URL":"http:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"}],"funder":[{"DOI":"10.13039\/100000011","name":"Howard Hughes Medical Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000011","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000865","name":"Bill and Melinda Gates Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000865","id-type":"DOI","asserted-by":"publisher"}]},{"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":["R35 GM118062"],"award-info":[{"award-number":["R35 GM118062"]}],"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":["R01 HL56067"],"award-info":[{"award-number":["R01 HL56067"]}],"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":["U01 AI142756"],"award-info":[{"award-number":["U01 AI142756"]}],"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":["RM1 HG009490"],"award-info":[{"award-number":["RM1 HG009490"]}],"id":[{"id":"10.13039\/100000002","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\/100006058","name":"Saint Baldricks Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006058","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007093","name":"Richard M. Schulze Family Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007093","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007249","name":"University of Minnesota","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007249","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"}]}],"content-domain":{"domain":["jidonline.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Investigative Dermatology"],"published-print":{"date-parts":[[2024,12]]},"DOI":"10.1016\/j.jid.2024.04.013","type":"journal-article","created":{"date-parts":[[2024,5,17]],"date-time":"2024-05-17T13:34:48Z","timestamp":1715952888000},"page":"2764-2777.e9","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":13,"title":["Twin Prime Editing Mediated Exon Skipping\/Reinsertion for Restored Collagen VII\u00a0Expression in Recessive Dystrophic Epidermolysis Bullosa"],"prefix":"10.1016","volume":"144","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6444-8643","authenticated-orcid":false,"given":"Benjamin J.","family":"Steinbeck","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2917-2060","authenticated-orcid":false,"given":"Xin D.","family":"Gao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4169-0015","authenticated-orcid":false,"given":"Amber N.","family":"McElroy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5461-5690","authenticated-orcid":false,"given":"Smriti","family":"Pandey","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9887-0391","authenticated-orcid":false,"given":"Jordan L.","family":"Doman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9557-217X","authenticated-orcid":false,"given":"Megan J.","family":"Riddle","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6320-1270","authenticated-orcid":false,"given":"Lily","family":"Xia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1998-5965","authenticated-orcid":false,"given":"Weili","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7314-2550","authenticated-orcid":false,"given":"Cindy R.","family":"Eide","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3632-343X","authenticated-orcid":false,"given":"Andre H.","family":"Lengert","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4953-7680","authenticated-orcid":false,"given":"Sang Won","family":"Han","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9608-9841","authenticated-orcid":false,"given":"Bruce R.","family":"Blazar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0240-9232","authenticated-orcid":false,"given":"Hans H.","family":"Wandall","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4279-8060","authenticated-orcid":false,"given":"Sally","family":"Dabelsteen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9943-7557","authenticated-orcid":false,"given":"David R.","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0957-4380","authenticated-orcid":false,"given":"Jakub","family":"Tolar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1790-0841","authenticated-orcid":false,"given":"Mark J.","family":"Osborn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jid.2024.04.013_bib1","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/s41587-021-01133-w","article-title":"Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing","volume":"40","author":"Anzalone","year":"2022","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.jid.2024.04.013_bib2","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.jid.2024.04.013_bib3","doi-asserted-by":"crossref","first-page":"250","DOI":"10.1016\/j.cell.2021.12.021","article-title":"Engineered virus-like particles for efficient in\u00a0vivo delivery of therapeutic proteins","volume":"185","author":"Banskota","year":"2022","journal-title":"Cell"},{"key":"10.1016\/j.jid.2024.04.013_bib4","article-title":"STRAIGHT-IN enables high-throughput targeting of large DNA payloads in human pluripotent stem cells","volume":"2","author":"Blanch-Asensio","year":"2022","journal-title":"Cell Rep Methods"},{"key":"10.1016\/j.jid.2024.04.013_bib5","doi-asserted-by":"crossref","first-page":"986","DOI":"10.1016\/j.ymthe.2019.03.007","article-title":"Clinically relevant correction of recessive dystrophic epidermolysis bullosa by dual sgRNA CRISPR\/Cas9-mediated gene editing","volume":"27","author":"Bonafont","year":"2019","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2024.04.013_bib6","doi-asserted-by":"crossref","first-page":"1302","DOI":"10.1038\/mt.2016.92","article-title":"Analysis of the functional consequences of targeted exon deletion in COL7A1 reveals prospects for dystrophic epidermolysis bullosa therapy","volume":"24","author":"Bornert","year":"2016","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2024.04.013_bib7","doi-asserted-by":"crossref","first-page":"242","DOI":"10.1016\/j.jpeds.2016.02.037","article-title":"Toxic epidermal necrolysis in recessive dystrophic epidermolysis bullosa following bone marrow transplantation","volume":"173","author":"Boull","year":"2016","journal-title":"J\u00a0Pediatr"},{"key":"10.1016\/j.jid.2024.04.013_bib8","doi-asserted-by":"crossref","first-page":"e379","DOI":"10.1038\/mtna.2016.87","article-title":"Antisense oligonucleotide-mediated exon skipping as a systemic therapeutic approach for recessive dystrophic epidermolysis bullosa","volume":"5","author":"Bremer","year":"2016","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.jid.2024.04.013_bib9","doi-asserted-by":"crossref","first-page":"318","DOI":"10.1038\/nature09328","article-title":"Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia","volume":"467","author":"Cavazzana-Calvo","year":"2010","journal-title":"Nature"},{"key":"10.1016\/j.jid.2024.04.013_bib10","doi-asserted-by":"crossref","first-page":"5635","DOI":"10.1016\/j.cell.2021.09.018","article-title":"Enhanced prime editing systems by manipulating cellular determinants of editing outcomes","volume":"184","author":"Chen","year":"2021","journal-title":"Cell"},{"key":"10.1016\/j.jid.2024.04.013_bib11","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1016\/S1525-0016(03)00168-0","article-title":"Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in\u00a0vivo","volume":"8","author":"Chen","year":"2003","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2024.04.013_bib12","doi-asserted-by":"crossref","first-page":"218","DOI":"10.1038\/s41587-021-01025-z","article-title":"Precise genomic deletions using paired prime editing","volume":"40","author":"Choi","year":"2022","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.jid.2024.04.013_bib13","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.2024.04.013_bib14","doi-asserted-by":"crossref","first-page":"1136","DOI":"10.1038\/s41467-019-09006-2","article-title":"CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations","volume":"10","author":"Cullot","year":"2019","journal-title":"Nat Commun"},{"key":"10.1016\/j.jid.2024.04.013_bib15","doi-asserted-by":"crossref","first-page":"1388","DOI":"10.1002\/stem.64","article-title":"Epithelial cells derived from human embryonic stem cells display p16INK4A senescence, hypermotility, and differentiation properties shared by many P63+ somatic cell types","volume":"27","author":"Dabelsteen","year":"2009","journal-title":"Stem Cells"},{"key":"10.1016\/j.jid.2024.04.013_bib16","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.jid.2024.04.013_bib17","doi-asserted-by":"crossref","first-page":"1436","DOI":"10.1128\/MCB.20.4.1436-1447.2000","article-title":"Human keratinocytes that express hTERT and also bypass a p16(INK4a)-enforced mechanism that limits life span become immortal yet retain normal growth and differentiation characteristics","volume":"20","author":"Dickson","year":"2000","journal-title":"Mol Cell Biol"},{"key":"10.1016\/j.jid.2024.04.013_bib18","doi-asserted-by":"crossref","first-page":"3983","DOI":"10.1016\/j.cell.2023.07.039","article-title":"Phage-assisted evolution and protein engineering yield compact, efficient prime editors","volume":"186","author":"Doman","year":"2023","journal-title":"Cell"},{"key":"10.1016\/j.jid.2024.04.013_bib19","doi-asserted-by":"crossref","first-page":"488","DOI":"10.1038\/s41587-022-01494-w","article-title":"Systematic discovery of recombinases for efficient integration of large DNA sequences into the human genome","volume":"41","author":"Durrant","year":"2023","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.jid.2024.04.013_bib20","doi-asserted-by":"crossref","first-page":"30248","DOI":"10.1074\/jbc.M109.045294","article-title":"Dominant-negative effects of COL7A1 mutations can be rescued by controlled overexpression of normal collagen VII","volume":"284","author":"Fritsch","year":"2009","journal-title":"J\u00a0Biol Chem"},{"key":"10.1016\/j.jid.2024.04.013_bib21","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A\u2022T to G\u2022C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.jid.2024.04.013_bib22","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1038\/JID.2015.364","article-title":"Lentiviral engineered fibroblasts expressing codon-optimized COL7A1 restore anchoring fibrils in RDEB","volume":"136","author":"Georgiadis","year":"2016","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib23","doi-asserted-by":"crossref","first-page":"2614","DOI":"10.1038\/sj.jid.5700435","article-title":"Targeted skipping of a single exon harboring a premature termination codon mutation: implications and potential for gene correction therapy for selective dystrophic epidermolysis bullosa patients","volume":"126","author":"Goto","year":"2006","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib24","doi-asserted-by":"crossref","first-page":"2211","DOI":"10.1056\/NEJMoa2206663","article-title":"Trial of Beremagene Geperpavec (B-VEC) for dystrophic epidermolysis bullosa","volume":"387","author":"Guide","year":"2022","journal-title":"N\u00a0Engl J Med"},{"key":"10.1016\/j.jid.2024.04.013_bib25","doi-asserted-by":"crossref","first-page":"780","DOI":"10.1038\/s41591-022-01737-y","article-title":"In\u00a0vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial","volume":"28","author":"Gurevich","year":"2022","journal-title":"Nat Med"},{"key":"10.1016\/j.jid.2024.04.013_bib26","doi-asserted-by":"crossref","first-page":"927","DOI":"10.1038\/s41591-018-0049-z","article-title":"CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response","volume":"24","author":"Haapaniemi","year":"2018","journal-title":"Nat Med"},{"key":"10.1016\/j.jid.2024.04.013_bib27","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\u00a0Clin Invest"},{"key":"10.1016\/j.jid.2024.04.013_bib28","doi-asserted-by":"crossref","first-page":"7705","DOI":"10.3390\/ijms21207705","article-title":"Nonsequential splicing events alter antisense-mediated exon skipping outcome in COL7A1","volume":"21","author":"Ham","year":"2020","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.jid.2024.04.013_bib29","doi-asserted-by":"crossref","first-page":"2664","DOI":"10.1016\/j.ymthe.2022.06.005","article-title":"Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa","volume":"30","author":"Hong","year":"2022","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2024.04.013_bib30","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":"Jack\u00f3w","year":"2016","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib31","doi-asserted-by":"crossref","first-page":"e408","DOI":"10.1002\/cpz1.408","article-title":"Differentiation of human induced pluripotent stem cells into keratinocytes","volume":"2","author":"Koch","year":"2022","journal-title":"Curr Protoc"},{"key":"10.1016\/j.jid.2024.04.013_bib32","doi-asserted-by":"crossref","first-page":"1985","DOI":"10.1016\/j.jid.2020.02.012","article-title":"Predictable CRISPR\/Cas9-mediated COL7A1 reframing for dystrophic epidermolysis bullosa","volume":"140","author":"Kocher","year":"2020","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib33","doi-asserted-by":"crossref","first-page":"496","DOI":"10.1016\/j.omtn.2019.09.011","article-title":"Improved double-nicking strategies for COL7A1-editing by\u00a0homologous recombination","volume":"18","author":"Kocher","year":"2019","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.jid.2024.04.013_bib34","doi-asserted-by":"crossref","first-page":"489","DOI":"10.1111\/j.1346-8138.2010.01008.x","article-title":"Exon 87 skipping of the COL7A1 gene in dominant dystrophic epidermolysis bullosa","volume":"38","author":"Koga","year":"2011","journal-title":"J\u00a0Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib35","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.jid.2024.04.013_bib36","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1038\/nbt.4192","article-title":"Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements","volume":"36","author":"Kosicki","year":"2018","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.jid.2024.04.013_bib37","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":"K\u00fchl","year":"2016","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib38","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1016\/j.jid.2016.11.038","article-title":"Correction of recessive dystrophic epidermolysis bullosa by transposon-mediated integration of COL7A1 in transplantable patient-derived primary keratinocytes","volume":"137","author":"Latella","year":"2017","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib39","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.143606","article-title":"Intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa patients","volume":"6","author":"Lee","year":"2021","journal-title":"JCI Insight"},{"key":"10.1016\/j.jid.2024.04.013_bib40","doi-asserted-by":"crossref","first-page":"895","DOI":"10.1038\/s41588-021-00838-7","article-title":"Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing","volume":"53","author":"Leibowitz","year":"2021","journal-title":"Nat Genet"},{"key":"10.1016\/j.jid.2024.04.013_bib41","doi-asserted-by":"crossref","first-page":"982","DOI":"10.1016\/j.tips.2018.09.001","article-title":"Precision medicine through antisense oligonucleotide-mediated exon skipping","volume":"39","author":"Li","year":"2018","journal-title":"Trends Pharmacol Sci"},{"key":"10.1016\/j.jid.2024.04.013_bib42","doi-asserted-by":"crossref","first-page":"4341","DOI":"10.3390\/ijms24054341","article-title":"COL7A1 editing via RNA trans-splicing in RDEB-derived skin equivalents","volume":"24","author":"Liemberger","year":"2023","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.jid.2024.04.013_bib43","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.126243","article-title":"Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa","volume":"4","author":"Lwin","year":"2019","journal-title":"JCI Insight"},{"key":"10.1016\/j.jid.2024.04.013_bib44","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.omtn.2018.01.009","article-title":"Deletion of a pathogenic mutation-containing exon of COL7A1 allows clonal gene editing correction of RDEB patient epidermal stem cells","volume":"11","author":"Menc\u00eda","year":"2018","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.jid.2024.04.013_bib45","doi-asserted-by":"crossref","first-page":"1807","DOI":"10.1038\/s41587-022-01377-0","article-title":"Frequent aneuploidy in primary human T cells after CRISPR-Cas9 cleavage","volume":"40","author":"Nahmad","year":"2022","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.jid.2024.04.013_bib46","doi-asserted-by":"crossref","DOI":"10.1016\/j.xjidi.2023.100191","article-title":"Cytosine deaminase base editing to restore COL7A1 in dystrophic epidermolysis bullosa human: murine skin model","volume":"3","author":"Naso","year":"2023","journal-title":"JID Innov"},{"key":"10.1016\/j.jid.2024.04.013_bib47","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1038\/s41587-021-01039-7","article-title":"Engineered pegRNAs improve prime editing efficiency","volume":"40","author":"Nelson","year":"2022","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.jid.2024.04.013_bib48","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1038\/mt.2010.249","article-title":"Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of alpha-L-iduronidase in mice with mucopolysaccharidosis type I","volume":"19","author":"Osborn","year":"2011","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2024.04.013_bib49","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1016\/j.jid.2019.07.701","article-title":"Base editor correction of COL7A1. in recessive dystrophic epidermolysis bullosa patient-derived fibroblasts and iPSCs","volume":"140","author":"Osborn","year":"2020","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib50","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.2024.04.013_bib73","doi-asserted-by":"crossref","DOI":"10.1038\/s41551-024-01227-1","article-title":"Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing","author":"Pandey","year":"2024","journal-title":"Nat Biomed Eng"},{"key":"10.1016\/j.jid.2024.04.013_bib51","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.2024.04.013_bib52","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.2024.04.013_bib53","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1007\/978-1-0716-0648-3_5","article-title":"Organotypic 3D skin models: human epidermal equivalent cultures from primary keratinocytes and immortalized keratinocyte cell lines","volume":"2154","author":"Rikken","year":"2020","journal-title":"Methods Mol Biol"},{"key":"10.1016\/j.jid.2024.04.013_bib54","doi-asserted-by":"crossref","first-page":"e934","DOI":"10.1111\/j.1365-2230.2009.03254.x","article-title":"A\u00a0novel de novo splice-site mutation in the COL7A1 gene in dominant dystrophic epidermolysis bullosa (DDEB): specific exon skipping could be a prognostic factor for DDEB pruriginosa","volume":"34","author":"Saito","year":"2009","journal-title":"Clin Exp Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib55","doi-asserted-by":"crossref","first-page":"1308","DOI":"10.1111\/bjd.13945","article-title":"A\u00a0COL7A1 variant leading to in-frame skipping of exon 15 attenuates disease severity in recessive dystrophic epidermolysis bullosa","volume":"173","author":"Schwieger-Briel","year":"2015","journal-title":"Br J Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib56","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-022-24184-8","article-title":"ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation","volume":"12","author":"Sheriff","year":"2022","journal-title":"Sci Rep"},{"key":"10.1016\/j.jid.2024.04.013_bib57","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-017-12041-y","article-title":"Immortalized N\/TERT keratinocytes as an alternative cell source in 3D human epidermal models","volume":"7","author":"Smits","year":"2017","journal-title":"Sci Rep"},{"key":"10.1016\/j.jid.2024.04.013_bib58","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1186\/s13023-022-02546-9","article-title":"Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa","volume":"17","author":"So","year":"2022","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.jid.2024.04.013_bib59","article-title":"Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectors [e-pub ahead of print]","author":"Suchy","year":"2024","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.jid.2024.04.013_bib60","doi-asserted-by":"crossref","first-page":"1711","DOI":"10.1016\/j.jid.2019.02.015","article-title":"Efficient gene reframing therapy for recessive dystrophic epidermolysis bullosa with CRISPR\/Cas9","volume":"139","author":"Takashima","year":"2019","journal-title":"J\u00a0Invest Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib61","doi-asserted-by":"crossref","first-page":"3685","DOI":"10.1038\/s41467-022-31322-3","article-title":"Frequency and mechanisms of LINE-1 retrotransposon insertions at CRISPR\/Cas9 sites","volume":"13","author":"Tao","year":"2022","journal-title":"Nat Commun"},{"key":"10.1016\/j.jid.2024.04.013_bib62","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\u00a0vivo gene therapy of recessive dystrophic epidermolysis bullosa","volume":"18","author":"Titeux","year":"2010","journal-title":"Mol Ther"},{"key":"10.1016\/j.jid.2024.04.013_bib63","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.jid.2024.04.013_bib64","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.2024.04.013_bib65","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1007\/978-1-61779-767-5_15","article-title":"Antisense-mediated exon skipping to reframe transcripts","volume":"867","author":"Turczynski","year":"2012","journal-title":"Methods Mol Biol"},{"key":"10.1016\/j.jid.2024.04.013_bib66","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.2024.04.013_bib67","doi-asserted-by":"crossref","first-page":"1247","DOI":"10.1111\/bjd.17943","article-title":"Revertant mosaic fibroblasts in recessive dystrophic epidermolysis bullosa","volume":"181","author":"Twaroski","year":"2019","journal-title":"Br J Dermatol"},{"key":"10.1016\/j.jid.2024.04.013_bib68","doi-asserted-by":"crossref","DOI":"10.3390\/ijms222212222","article-title":"Therapeutic prospects of exon skipping for epidermolysis bullosa","volume":"22","author":"Vermeer","year":"2021","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.jid.2024.04.013_bib69","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.2024.04.013_bib70","doi-asserted-by":"crossref","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"},{"key":"10.1016\/j.jid.2024.04.013_bib71","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1186\/1472-6750-13-87","article-title":"Accuracy and efficiency define Bxb1 integrase as the best of fifteen candidate serine recombinases for the integration of DNA into the human genome","volume":"13","author":"Xu","year":"2013","journal-title":"BMC Biotechnol"},{"key":"10.1016\/j.jid.2024.04.013_bib72","doi-asserted-by":"crossref","first-page":"500","DOI":"10.1038\/s41587-022-01527-4","article-title":"Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases","volume":"41","author":"Yarnall","year":"2023","journal-title":"Nat Biotechnol"}],"container-title":["Journal of Investigative Dermatology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0022202X24003725?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0022202X24003725?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,12,1]],"date-time":"2025-12-01T01:05:40Z","timestamp":1764551140000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0022202X24003725"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,12]]},"references-count":73,"journal-issue":{"issue":"12","published-print":{"date-parts":[[2024,12]]}},"alternative-id":["S0022202X24003725"],"URL":"https:\/\/doi.org\/10.1016\/j.jid.2024.04.013","relation":{},"ISSN":["0022-202X"],"issn-type":[{"value":"0022-202X","type":"print"}],"subject":[],"published":{"date-parts":[[2024,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Twin Prime Editing Mediated Exon Skipping\/Reinsertion for Restored Collagen VII\u00a0Expression in Recessive Dystrophic 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.2024.04.013","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Authors. Published by Elsevier, Inc. on behalf of the Society for Investigative Dermatology.","name":"copyright","label":"Copyright"}]}}