{"id":"W1979255161","doi":"10.1038/gt.2011.94","title":"Encapsulated engineered myoblasts can cure Hurler syndrome: preclinical experiments in the mouse model","year":2011,"lang":"en","type":"article","venue":"Gene Therapy","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":18,"is_retracted":false,"has_abstract":false,"ca_institutions":"McMaster University","funders":"","keywords":"Enzyme replacement therapy; Mucopolysaccharidosis I; Mucopolysaccharidosis type I; Mucopolysaccharidosis; Transplantation; Hurler syndrome; Glycosaminoglycan; Lysosomal storage disease; Mucopolysaccharidosis type II; Hunter syndrome; Biology; Stem cell; Pathology; Medicine; Immunology; Cell biology; Disease; Internal medicine; Biochemistry","routes":{"ca_aff":true,"ca_fund":false,"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":[0.001216473,0.00137393,0.0008759826,0.001290448,0.0004326653,0.0005930156,0.0009269522,0.002212708,0.001659307],"category_scores_gemma":[0.0005010274,0.0004064502,0.0009053124,0.0003503369,0.001055934,0.0008401378,0.0004070186,0.001855599,0.0005063721],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004369619,"about_ca_system_score_gemma":0.0004748357,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001519355,"about_ca_topic_score_gemma":0.00218388,"domain_scores_codex":[0.9994845,0.0001203837,0.00004579185,0.00009953257,0.0001502285,0.00009947544],"domain_scores_gemma":[0.9994833,0.0001599508,0.0001419433,0.00006688318,0.00003713337,0.0001106795],"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":[0.001094345,0.0008192507,0.0001297288,0.0001265139,0.00004946836,0.0002301779,0.00008175251,0.0004780545,0.9935006,0.0004333255,0.0001995962,0.002857093],"study_design_scores_gemma":[0.0005006598,0.006335373,0.0009361918,0.00003637752,0.0001472106,0.0002962958,0.0000557967,0.001307371,0.9883034,0.0002034132,0.001859005,0.00001877672],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9850482,0.003705496,0.006114559,0.0004818119,0.0002112032,0.0002572043,0.0008146157,0.0003798414,0.002987095],"genre_scores_gemma":[0.9797657,0.003817646,0.006460117,0.0001587967,0.00006532686,0.0002268054,0.0009679183,0.0001499838,0.008387635],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002212708,"threshold_uncertainty_score":0.006433368,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1148679479642486,"score_gpt":0.3499602956335889,"score_spread":0.2350923476693403,"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."}}