{"id":"W2028390112","doi":"10.1038/mt.2013.4","title":"Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases","year":2013,"lang":"en","type":"letter","venue":"Molecular Therapy","topic":"Pluripotent Stem Cells Research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":12,"is_retracted":false,"has_abstract":true,"ca_institutions":"Biotechnology Research Institute; Université Laval; Université de Montréal; Polytechnique Montréal; Children's Hospital of Eastern Ontario; University of Ottawa","funders":"National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Diabetes and Digestive and Kidney Diseases; National Heart, Lung, and Blood Institute; School of Medicine, Indiana University; Medical School, University of Michigan; School of Medicine, Stanford University; State Key Laboratory of Oncology in South China; Peking Union Medical College; Université de Montréal; Chinese Academy of Medical Sciences; National Center for Advancing Translational Sciences; Medical Research Council; Leids Universitair Medisch Centrum; Perelman School of Medicine, University of Pennsylvania; Universiteit Leiden; National Institutes of Health; Rosetrees Trust; University of Oxford; Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas; Directorate for Biological Sciences; University of Ottawa; University of California, San Francisco; Nationwide Children's Hospital; National Institute of Neurological Disorders and Stroke; University of Pittsburgh; Université Laval; University of Washington; National Center of Neurology and Psychiatry; Ohio State University; Sichuan University; Newcastle University; Sun Yat-sen University; Howard Hughes Medical Institute; Children's Hospital of Philadelphia; Wellcome Trust; University of Pennsylvania; Vrije Universiteit Brussel; National Institute for Health and Care Research; Massachusetts General Hospital","keywords":"Genetic enhancement; Induced pluripotent stem cell; Biology; Genome editing; Zinc finger nuclease; Stem cell; Embryonic stem cell; Reprogramming; Cell therapy; Bioinformatics; Biotechnology; Genetics; Medicine; Computational biology; Gene; CRISPR","routes":{"ca_aff":true,"ca_fund":true,"ca_venue":false,"about_ca":true,"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":[0.01372575,0.001148515,0.002007319,0.001316925,0.002119292,0.006217655,0.003830634,0.02245371,0.009619954],"category_scores_gemma":[0.04722312,0.0005557858,0.001125268,0.001025837,0.005265601,0.009527856,0.002298478,0.02662218,0.004197071],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.002450264,"about_ca_system_score_gemma":0.006074438,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001054747,"about_ca_topic_score_gemma":0.002198857,"domain_scores_codex":[0.9938021,0.001870682,0.0009662298,0.0006315251,0.00209251,0.0006369337],"domain_scores_gemma":[0.9317327,0.03489723,0.003613096,0.001621075,0.01712936,0.0110065],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"not_applicable","study_design_gemma":"not_applicable","study_design_scores_codex":[0.00004988298,0.00002278208,0.00007231932,0.0002942733,0.00001701939,0.0004182594,0.00008111368,0.00002822071,0.0001303397,0.002807104,0.9764884,0.0195904],"study_design_scores_gemma":[0.00005177812,0.00004547736,0.0002538951,0.0005378446,0.00004171172,0.0008732899,0.0002859636,0.0001139728,0.0002110128,0.004537662,0.9930131,0.00003425757],"study_design_candidate":"not_applicable","study_design_consensus":"not_applicable","genre_codex":"commentary","genre_gemma":"commentary","genre_scores_codex":[0.0001684632,0.00965415,0.0003813412,0.7838006,0.2052251,0.000009530602,0.00003651371,0.00004858649,0.0006757713],"genre_scores_gemma":[0.00305103,0.01505319,0.001096652,0.6016927,0.3746427,0.00004457337,0.00006887317,0.00008602998,0.004264201],"genre_candidate":"commentary","genre_consensus":"commentary","teacher_disagreement_score":0.02245371,"threshold_uncertainty_score":0.07258964,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03223635630898778,"score_gpt":0.290728186196045,"score_spread":0.2584918298870572,"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."}}