{"id":"W2948829110","doi":"10.1172/jci.insight.126243","title":"Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa","year":2019,"lang":"en","type":"article","venue":"JCI Insight","topic":"Skin and Cellular Biology Research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":90,"is_retracted":false,"has_abstract":true,"ca_institutions":"St. Thomas Hospital","funders":"Menzies Centre for Australian Studies, King's College London, University of London; King's College London; National Institute for Health and Care Research; Fondation René Touraine; Biotechnology and Biological Sciences Research Council; Cure EB; Institut des maladies génétiques Imagine; Dystrophic Epidermolysis Bullosa Research Association","keywords":"Genetic enhancement; Epidermolysis bullosa; Medicine; Fibroblast; Dermatology; Gene; Junctional epidermolysis bullosa (veterinary medicine); Cancer research; Genetics; Mutation; Biology; Cell culture","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001419261,0.0005351143,0.0007550921,0.0003143345,0.0001738547,0.00046049,0.0002825899,0.0006242885,0.001255166],"category_scores_gemma":[0.001064705,0.00008904644,0.0006942406,0.0001314177,0.0003055467,0.0003199793,0.0001347617,0.0008403471,0.0001742198],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003974057,"about_ca_system_score_gemma":0.0003751034,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002865725,"about_ca_topic_score_gemma":0.0003922496,"domain_scores_codex":[0.9993944,0.0002334507,0.00005162359,0.00007134955,0.0001351467,0.0001140702],"domain_scores_gemma":[0.9991953,0.0002251777,0.0002975335,0.00004364839,0.00009369415,0.0001446742],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.2727217,0.04349411,0.02364128,0.001629083,0.001102547,0.0008212557,0.0002905916,0.002911711,0.3207396,0.0003386087,0.001813319,0.3304961],"study_design_scores_gemma":[0.01625881,0.8301558,0.05632225,0.0001225064,0.0009767413,0.0007459931,0.0001067056,0.002410323,0.09018713,0.0002236921,0.002455787,0.00003432183],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9945443,0.003112984,0.0006400274,0.00008219026,0.000039485,0.0004009576,0.0003084057,0.00003717237,0.0008344315],"genre_scores_gemma":[0.9978046,0.0006255991,0.0004395597,0.0000842982,0.00003794527,0.0002599258,0.0002786149,0.000006714442,0.0004627589],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001419261,"threshold_uncertainty_score":0.007505834,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01433801790018227,"score_gpt":0.2716535464472349,"score_spread":0.2573155285470527,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}