{"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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0003650739,0.0004418606,0.0002916167,0.00006363893,0.0003755111,0.0002020841,0.001547211,0.0005312435,0.00003549307],"category_scores_gemma":[0.00004264041,0.0002829051,0.000645058,0.00007441241,0.0002269883,0.00001065491,0.00007054617,0.0004206581,0.000007527561],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00005499958,"about_ca_system_score_gemma":0.0002451562,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00002707179,"about_ca_topic_score_gemma":0.000004563077,"domain_scores_codex":[0.9975694,0.0003044625,0.0004045421,0.0007408163,0.0003822051,0.0005985794],"domain_scores_gemma":[0.998075,0.0001006185,0.0002313796,0.001168446,0.0003508597,0.00007369437],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0005587498,0.00007764932,0.00004987013,0.00002992157,0.001092989,0.000006285653,0.0000855572,0.0002101324,0.8293642,0.00003274357,0.1555232,0.01296872],"study_design_scores_gemma":[0.001733844,0.0004831514,0.00006619519,0.000008327775,0.00004550291,0.00001779595,0.00002482838,0.001223914,0.09729639,0.0004452052,0.8982671,0.0003877879],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"commentary","genre_gemma":"commentary","genre_scores_codex":[0.214177,0.05343355,0.08421636,0.6292824,0.001727853,0.01452625,0.002270283,0.000088528,0.000277805],"genre_scores_gemma":[0.1080035,0.02320757,0.00544438,0.8029292,0.01856691,0.01185778,0.02270181,0.0009122234,0.006376633],"genre_candidate":"commentary","genre_consensus":"commentary","teacher_disagreement_score":0.7427439,"threshold_uncertainty_score":0.9999623,"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."}}