{"id":"W1992657824","doi":"10.4236/ojmc.2013.31001","title":"New Gene Therapy Strategies for the Deletion of Exon 44 of Dystrophin Gene Based on Gene Editing by TALENs","year":2013,"lang":"en","type":"article","venue":"Open Journal of Medicinal Chemistry","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Centre hospitalier de l'Université Laval; University of Waterloo","funders":"Division of Graduate Education; Priority Academic Program Development of Jiangsu Higher Education Institutions; National Natural Science Foundation of China","keywords":"Transcription activator-like effector nuclease; Genetic enhancement; Duchenne muscular dystrophy; Muscular dystrophy; Genome editing; Dystrophin; Genetics; Exon; Gene; Exon skipping; Biology; Bioinformatics; Medicine; Computational biology; CRISPR; 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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.000278988,0.0005604556,0.0005146774,0.000566853,0.0002913384,0.0004389909,0.0005347775,0.0006406161,0.0008673841],"category_scores_gemma":[0.0001359444,0.000258778,0.0005904332,0.0003068481,0.0004980974,0.0006597105,0.0005006269,0.0009339316,0.0002818136],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002621958,"about_ca_system_score_gemma":0.0001599987,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002310471,"about_ca_topic_score_gemma":0.0003552692,"domain_scores_codex":[0.9997904,0.00002710416,0.00001700489,0.00005317812,0.00007981413,0.00003250484],"domain_scores_gemma":[0.9999498,0.00001074622,0.00001931814,0.000005047518,0.000005930732,0.000009102956],"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":[0.00006401964,0.0001113392,0.00006700863,0.000377526,0.00003555843,0.0003371291,0.0001217589,0.0006129254,0.9749402,0.003741957,0.0003914945,0.01919896],"study_design_scores_gemma":[0.0000649914,0.0007089487,0.0004218527,0.00003100044,0.00008386563,0.001295503,0.00004570248,0.003082142,0.9656394,0.0008377031,0.02774663,0.00004221016],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.5933194,0.06483952,0.3170227,0.001483716,0.001007279,0.0007680191,0.0004231522,0.00123987,0.01989624],"genre_scores_gemma":[0.8789066,0.03262908,0.07397547,0.000679661,0.00009693026,0.000389898,0.0003516075,0.00009317568,0.01287759],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0008673841,"threshold_uncertainty_score":0.002901733,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02416526063437841,"score_gpt":0.3115331343893655,"score_spread":0.2873678737549871,"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."}}