{"id":"W2095208613","doi":"10.1089/hum.2008.162","title":"Efficient and Fast Functional Screening of Microdystrophin Constructs <i>In Vivo</i> and <i>In Vitro</i> for Therapy of Duchenne Muscular Dystrophy","year":2009,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":16,"is_retracted":false,"has_abstract":true,"ca_institutions":"Montreal Neurological Institute and Hospital; McGill University","funders":"","keywords":"Duchenne muscular dystrophy; Dystrophin; Exon skipping; Muscular dystrophy; Genetic enhancement; In silico; Transgene; Biology; Computational biology; Exon; In vivo; Gene; In vitro; Bioinformatics; Genetics; Alternative splicing","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":[0.0001341755,0.0001434072,0.0002350344,0.00007324692,0.00004590077,0.000004384785,0.00006889103,0.00009876507,0.000007237848],"category_scores_gemma":[0.000005014032,0.0001351283,0.0000648433,0.00005948286,0.0001840201,0.000002337979,0.00001564655,0.00005225351,3.833736e-8],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000003171501,"about_ca_system_score_gemma":0.00002007816,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001923318,"about_ca_topic_score_gemma":0.000006138344,"domain_scores_codex":[0.9991943,0.00004668425,0.0002392244,0.0002859448,0.00005885112,0.0001750056],"domain_scores_gemma":[0.999648,0.00001318145,0.00009859635,0.0001614916,0.00004604843,0.00003267798],"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":[0.002296718,0.0001426854,0.0007106764,0.00001096486,0.00003142094,6.65684e-7,0.0000701262,0.0005486661,0.9916306,0.00008228944,0.00001770481,0.004457448],"study_design_scores_gemma":[0.005413267,0.0008164119,0.04088883,0.00001207595,0.000003628566,0.000006071939,0.00006433,0.00009495167,0.9519982,0.0003209265,0.0002343526,0.0001469961],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.994673,0.004466593,0.0004419131,0.00004049625,0.00002312669,0.0002905196,0.00003125622,0.000003055997,0.00003002501],"genre_scores_gemma":[0.997943,0.0006611059,0.001085383,0.0001938829,0.00004298861,0.00001350232,0.00004128342,0.00001077562,0.000008016168],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.04017815,"threshold_uncertainty_score":0.551037,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0117491474215175,"score_gpt":0.2323797598550573,"score_spread":0.2206306124335398,"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."}}