{"id":"W2094208378","doi":"10.1111/j.1651-2227.2002.tb03119.x","title":"Non‐viral transfer approaches for the gene therapy of mucopolysaccharidosis type II (Hunter syndrome)","year":2002,"lang":"en","type":"article","venue":"Acta Paediatrica","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":25,"is_retracted":false,"has_abstract":true,"ca_institutions":"McMaster University","funders":"","keywords":"Mucopolysaccharidosis type II; Genetic enhancement; Enzyme replacement therapy; Hunter syndrome; Viral vector; Medicine; Cell culture; Fibroblast; Heterologous; Transplantation; Cell therapy; Molecular biology; Cell; Virology; Biology; Gene; Internal medicine; Biochemistry; Disease; Genetics; Recombinant DNA","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":["insufficient_payload"],"consensus_categories":[],"category_scores_codex":[0.000201214,0.0001844806,0.0003419788,0.0001416901,0.0001730938,0.00001586614,0.0003206959,0.0001364274,0.001079562],"category_scores_gemma":[0.00008572236,0.000116175,0.0002600539,0.0005009458,0.0001157889,0.0000859752,0.00005138325,0.0002218653,0.00003461388],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000039896,"about_ca_system_score_gemma":0.00005183789,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005809435,"about_ca_topic_score_gemma":0.000002097335,"domain_scores_codex":[0.9985663,0.00002972004,0.0002808234,0.0002913672,0.0004298007,0.0004019407],"domain_scores_gemma":[0.999046,0.0002131265,0.00004126965,0.0004641578,0.0001248023,0.000110646],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.008653995,0.01247835,0.09493652,0.00139017,0.005939772,0.0002373667,0.02779375,0.00007741836,0.1765695,0.0005985359,0.2103256,0.460999],"study_design_scores_gemma":[0.07425227,0.0442165,0.2397399,0.0002040015,0.00358806,0.001075392,0.004448541,0.06939716,0.2383777,0.0007563009,0.3194888,0.004455335],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9851062,0.007033582,0.0004076797,0.004689524,0.0001615932,0.001461291,0.00002840502,0.00003982386,0.001071842],"genre_scores_gemma":[0.9945394,0.00342096,0.0002678307,0.0003123915,0.0002239712,0.0001320704,0.00001466712,0.00004847894,0.001040183],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.4565436,"threshold_uncertainty_score":0.9998336,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06502861582427653,"score_gpt":0.2709021483875793,"score_spread":0.2058735325633028,"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."}}