{"id":"W2194139052","doi":"10.1371/journal.pone.0143846","title":"Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)","year":2015,"lang":"en","type":"article","venue":"PLoS ONE","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":69,"is_retracted":false,"has_abstract":true,"ca_institutions":"QLT (Canada); McGill University Health Centre","funders":"National Institute for Health and Care Research; Canadian Institutes of Health Research; National Eye Institute; Johns Hopkins Bloomberg School of Public Health; National Human Genome Research Institute; Johns Hopkins University","keywords":"RPE65; Retinitis pigmentosa; Lecithin; Pigment; Retinal; Ophthalmology; Acyltransferase; Biology; Cell biology; Medicine; Chemistry; Biochemistry; Retinal pigment epithelium; Enzyme","routes":{"ca_aff":true,"ca_fund":true,"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":[0.001201991,0.0007300201,0.0007044241,0.0002399988,0.000192671,0.0005546253,0.0007714677,0.0008210959,0.001747756],"category_scores_gemma":[0.0005359662,0.0002377615,0.0006211773,0.0001698149,0.0006129435,0.0005321438,0.0001990525,0.001652159,0.0004641993],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003136362,"about_ca_system_score_gemma":0.0006576655,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003451864,"about_ca_topic_score_gemma":0.000429995,"domain_scores_codex":[0.9997219,0.0001072168,0.00001579223,0.00005537186,0.00005697742,0.00004262986],"domain_scores_gemma":[0.9995067,0.00009358161,0.0001714812,0.00005393209,0.00005641791,0.0001178531],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.08161768,0.02262898,0.00228983,0.001525291,0.0005015084,0.0005994675,0.0002659762,0.001364432,0.7945276,0.0002608347,0.002083334,0.092335],"study_design_scores_gemma":[0.06759919,0.6697336,0.01378023,0.0001469115,0.0008613238,0.00212924,0.0001706607,0.003720903,0.231145,0.0001778416,0.01048714,0.00004792454],"study_design_candidate":"randomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9933434,0.002094814,0.002551309,0.0002028736,0.00006918945,0.0005533327,0.000515205,0.0001098579,0.0005600851],"genre_scores_gemma":[0.9930716,0.001413563,0.003134677,0.00023455,0.0000475699,0.0005501304,0.000676047,0.000029006,0.0008427871],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001747756,"threshold_uncertainty_score":0.006356776,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04894217223669953,"score_gpt":0.2531391234397906,"score_spread":0.204196951203091,"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."}}