{"id":"W2587028347","doi":"10.1038/nbt.3787","title":"Industrializing rare disease therapy discovery and development","year":2017,"lang":"en","type":"letter","venue":"Nature Biotechnology","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":30,"is_retracted":false,"has_abstract":false,"ca_institutions":"","funders":"National Institute of Neurological Disorders and Stroke; National Institutes of Health; Rare Disease Foundation","keywords":"Disease; Rare disease; Medicine; Internal medicine","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.004118956,0.0006859187,0.0006750433,0.0004665891,0.002426257,0.00407645,0.001360779,0.03082593,0.004045216],"category_scores_gemma":[0.01372324,0.0005399799,0.0008143286,0.0003068469,0.005261824,0.002474636,0.003216181,0.02922896,0.003027747],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00286506,"about_ca_system_score_gemma":0.002606946,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001020282,"about_ca_topic_score_gemma":0.002163949,"domain_scores_codex":[0.9973264,0.0005011393,0.0001562191,0.0003068069,0.001307978,0.0004013958],"domain_scores_gemma":[0.9942024,0.003958216,0.0002293115,0.0002922663,0.0005907431,0.0007270615],"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":[0.000129179,0.0001018674,0.000510133,0.0001188632,0.00002962844,0.002183533,0.000119016,0.0003527008,0.002071653,0.05007636,0.8968707,0.04743635],"study_design_scores_gemma":[0.0001912692,0.0001148965,0.0002621566,0.00009760173,0.00002820982,0.001306767,0.0001221915,0.0008985977,0.003112961,0.08103521,0.9127953,0.00003485943],"study_design_candidate":"not_applicable","study_design_consensus":"not_applicable","genre_codex":"commentary","genre_gemma":"commentary","genre_scores_codex":[0.001137968,0.007386569,0.002758392,0.9460059,0.03090881,0.00003281591,0.00005173302,0.0001197545,0.01159804],"genre_scores_gemma":[0.0366662,0.01040142,0.004059399,0.8603272,0.06114216,0.0001875527,0.00009920934,0.0000778337,0.02703895],"genre_candidate":"commentary","genre_consensus":"commentary","teacher_disagreement_score":0.03082593,"threshold_uncertainty_score":0.02178335,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01228337264999695,"score_gpt":0.2887777277612916,"score_spread":0.2764943551112946,"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."}}