{"id":"W4398171854","doi":"10.3390/cells13100877","title":"Heterologous HSPC Transplantation Rescues Neuroinflammation and Ameliorates Peripheral Manifestations in the Mouse Model of Lysosomal Transmembrane Enzyme Deficiency, MPS IIIC","year":2024,"lang":"en","type":"article","venue":"Cells","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":4,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University; Université de Montréal; Centre Hospitalier Universitaire Sainte-Justine","funders":"Canadian Glycomics Network; Canadian Institutes of Health Research; Kyowa Hakko Kirin; Sanfilippo Children's Foundation","keywords":"Neuroinflammation; Microglia; Heparan sulfate; Lysosomal storage disease; Enzyme replacement therapy; Transplantation; Astrogliosis; Biology; Cell biology; Immunology; Pathology; Medicine; Neuroscience; Internal medicine; Central nervous system; Biochemistry; Inflammation; Disease; Glycosaminoglycan","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":[],"consensus_categories":[],"category_scores_codex":[0.0002465653,0.000123297,0.0001593105,0.0001981583,0.00006150533,0.00005218903,0.0001019153,0.00007223796,0.00002532277],"category_scores_gemma":[0.00001658075,0.00009068879,0.00005745393,0.0002769975,0.0001303422,0.0001778364,0.000007670906,0.0002188836,0.000007206217],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002809148,"about_ca_system_score_gemma":0.00007677059,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00009691797,"about_ca_topic_score_gemma":0.0001407778,"domain_scores_codex":[0.9988374,0.0001127331,0.0002819709,0.0002405179,0.0003165642,0.0002108253],"domain_scores_gemma":[0.9996023,0.0001203588,0.00002669361,0.0001566163,0.00004920188,0.00004478812],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"simulation_or_modeling","study_design_scores_codex":[0.0002936625,0.0001587252,0.0005328757,0.001349569,0.00001589644,0.0002241001,0.01685682,0.01790486,0.9603326,0.0006678009,0.00005317688,0.001609879],"study_design_scores_gemma":[0.002985805,0.001147759,0.01367466,0.000253675,0.0002538896,0.0004257861,0.002794852,0.6797478,0.2964812,0.001799301,0.00006123611,0.0003740369],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9951004,0.0009145314,0.002045147,0.0008066581,0.0000297262,0.0005220026,0.0000389013,0.0000445652,0.0004980868],"genre_scores_gemma":[0.9983804,0.0009238244,0.0002585045,0.00007220036,0.0000211554,0.00003289777,0.00006302197,0.00002008377,0.000227977],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.6638514,"threshold_uncertainty_score":0.3698182,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02335333393713621,"score_gpt":0.2769768670176262,"score_spread":0.25362353308049,"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."}}