{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001984873,0.0001748419,0.000278179,0.00008253482,0.00006267276,0.00002578483,0.0001709109,0.00022952,0.00003754439],"category_scores_gemma":[0.00007365781,0.0001375594,0.0001250778,0.00009574407,0.00009276377,0.000005664358,0.0001022151,0.000118556,0.00002029047],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001251559,"about_ca_system_score_gemma":0.0000419391,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00002531821,"about_ca_topic_score_gemma":0.00001050219,"domain_scores_codex":[0.9987795,0.0001180187,0.0002366132,0.000453759,0.00009409454,0.0003179714],"domain_scores_gemma":[0.9994189,0.00007332112,0.000069031,0.0003203402,0.00005637105,0.00006210306],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.0008718251,0.0001596786,0.08336835,0.00001957741,0.0001542557,0.00003215201,0.0001141007,0.00000995949,0.9089317,0.00009396017,0.00009360915,0.006150773],"study_design_scores_gemma":[0.008898321,0.002376484,0.3375123,0.00004478365,0.0000188327,0.00009393875,0.00006831368,0.00007033286,0.6230049,0.0001681557,0.02714841,0.0005952575],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9969871,0.001356396,0.0001588483,0.000314398,0.00009462893,0.0005485918,0.00001893677,0.000007684598,0.0005133824],"genre_scores_gemma":[0.99624,0.0008708791,0.0002287738,0.00007654843,0.00009304367,0.00003799004,0.000120194,0.00002359388,0.002308978],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2859269,"threshold_uncertainty_score":0.5609508,"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."}}