{"id":"W2619708749","doi":"10.1016/j.bmcl.2017.05.086","title":"A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols","year":2017,"lang":"en","type":"article","venue":"Bioorganic & Medicinal Chemistry Letters","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":16,"is_retracted":false,"has_abstract":false,"ca_institutions":"University of British Columbia","funders":"Canadian Glycomics Network; Austrian Science Fund","keywords":"Chemistry; Gangliosidosis; Lysosomal storage disease; Hydroxymethyl; Chaperone (clinical); Biochemistry; Enzyme; Stereochemistry","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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow","insufficient_payload"],"consensus_categories":[],"category_scores_codex":[0.000418711,0.0004703196,0.0009114225,0.0001217427,0.0004613989,0.00005640776,0.0009334285,0.000408426,0.00329644],"category_scores_gemma":[0.001135495,0.0004121412,0.0002670046,0.0003798598,0.0006622212,0.0001664387,0.0002458374,0.000696521,0.00004594759],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003209293,"about_ca_system_score_gemma":0.0003815226,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002207693,"about_ca_topic_score_gemma":0.000004549772,"domain_scores_codex":[0.9966122,0.00004725018,0.0007672138,0.0007871639,0.0009660467,0.0008201327],"domain_scores_gemma":[0.9974115,0.0001602062,0.0005253724,0.0009346794,0.0002617034,0.0007064615],"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":[0.0008182921,0.0002801398,0.006898099,0.0003979864,0.0005707104,0.0004329488,0.0001887474,4.039708e-7,0.982266,0.00001152897,0.006862877,0.00127223],"study_design_scores_gemma":[0.01043474,0.0004723273,0.02486076,0.0004146414,0.0009268082,0.0003446728,0.0001533309,0.00006749255,0.9601008,0.0000259044,0.001719659,0.0004789066],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9865184,0.0008965678,0.0002395887,0.009618235,0.0003058816,0.001065098,0.00002910448,0.0001186001,0.001208483],"genre_scores_gemma":[0.9967098,0.0001204444,0.0005075256,0.0005969927,0.0007084017,0.00003815867,0.0002060635,0.00008002976,0.001032577],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02216527,"threshold_uncertainty_score":0.999833,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05077207427793002,"score_gpt":0.3521340037549983,"score_spread":0.3013619294770683,"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."}}