{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003415244,0.0001961682,0.0002870993,0.0001179649,0.00005066055,0.00001445707,0.0003504351,0.0001590241,0.0004242279],"category_scores_gemma":[0.00003728832,0.000131208,0.0000992115,0.0002914149,0.00007372243,0.00005447237,0.00003962308,0.0003708936,0.0000479546],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000675101,"about_ca_system_score_gemma":0.0001245713,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002169927,"about_ca_topic_score_gemma":0.00002441219,"domain_scores_codex":[0.9983487,0.0001265404,0.0002987741,0.0003186819,0.0004735019,0.0004338473],"domain_scores_gemma":[0.9991361,0.00003062069,0.00003957313,0.0005910329,0.00006958901,0.0001330625],"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":[0.005705141,0.008057474,0.0269291,0.00008724113,0.001001206,0.001150829,0.1107943,0.001333879,0.8235476,0.0002213762,0.005408785,0.01576308],"study_design_scores_gemma":[0.03959992,0.006980363,0.1910972,0.0001677081,0.0001031367,0.0006096924,0.003506278,0.04902274,0.703029,0.001656582,0.002412335,0.001815001],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9966443,0.001148102,0.000109565,0.000502456,0.00004488361,0.0008587471,0.00001030293,0.00006200597,0.0006196246],"genre_scores_gemma":[0.9966469,0.0006136389,0.0006585449,0.0007535986,0.00003425874,0.0001392188,0.00003155844,0.0000471966,0.00107505],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1641681,"threshold_uncertainty_score":0.5350506,"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."}}