{"id":"W4407357821","doi":"10.1016/j.omtm.2025.101432","title":"Cathepsin B inhibition blocks amyloidogenesis in the mouse models of neurological lysosomal diseases MPS IIIC and sialidosis","year":2025,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University; Centre Hospitalier Universitaire Sainte-Justine","funders":"Institute of Genetics; Fundação de Amparo à Pesquisa do Estado de São Paulo; Sanfilippo Children's Foundation; Sanfilippo Research Foundation; Canadian Glycomics Network; Canadian Institutes of Health Research","keywords":"Mucopolysaccharidosis; Mucopolysaccharidosis type I; Mucolipidosis; Lysosomal storage disease; Cathepsin D; Lysosome; Cathepsin; Medicine; Chemistry; Pathology; Biochemistry; Enzyme; Enzyme replacement therapy; Disease","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":[0.000243861,0.0008079496,0.0005100291,0.0007757761,0.0002648344,0.0004383547,0.0004507637,0.0007142915,0.001218659],"category_scores_gemma":[0.0001716329,0.0003872193,0.0005877863,0.0003536877,0.0005240883,0.0002300209,0.000279517,0.001250631,0.0003732128],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005319904,"about_ca_system_score_gemma":0.0005248852,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.002571725,"about_ca_topic_score_gemma":0.004223785,"domain_scores_codex":[0.999619,0.00005088278,0.00006063567,0.00006972902,0.00009079179,0.0001088933],"domain_scores_gemma":[0.9996027,0.00004148985,0.0001334783,0.00004118708,0.00003054293,0.0001506036],"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":[0.000490962,0.0001275356,0.0001913533,0.00004419846,0.00002110722,0.0001133376,0.00002844372,0.00009701344,0.9979486,0.0001134652,0.00009008132,0.0007338976],"study_design_scores_gemma":[0.0001072583,0.001363066,0.005460722,0.0000235137,0.00004695568,0.00046795,0.00007519048,0.0009580836,0.9897989,0.0001206226,0.001562609,0.0000152022],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9942264,0.001214782,0.001131752,0.0001732158,0.00004801125,0.00005607856,0.001337836,0.0002159372,0.001596018],"genre_scores_gemma":[0.9917658,0.001417127,0.001928916,0.00009554212,0.00001360465,0.0001311532,0.001186558,0.00007858683,0.003382682],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002571725,"threshold_uncertainty_score":0.005113482,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.08199099614081866,"score_gpt":0.4243183676932385,"score_spread":0.3423273715524198,"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."}}