{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0009029392,0.0001904613,0.0003346064,0.00003138131,0.00007609129,0.00004266215,0.0008695677,0.000145295,0.000322936],"category_scores_gemma":[0.0001057787,0.0001299089,0.0001670243,0.0000883716,0.0001471927,0.00001694938,0.00006064626,0.0002304441,6.989972e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000291233,"about_ca_system_score_gemma":0.0006608979,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00007202302,"about_ca_topic_score_gemma":0.000003443477,"domain_scores_codex":[0.998385,0.00006879824,0.0005687749,0.000205983,0.0005162898,0.0002552004],"domain_scores_gemma":[0.9982799,0.00008821639,0.000633544,0.0003725109,0.0004682734,0.0001575432],"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":[0.00280373,0.00009513021,0.00004941831,0.00004517249,0.000145984,0.000003227062,0.00003515766,0.0006481824,0.9772868,7.590571e-7,0.008129409,0.01075709],"study_design_scores_gemma":[0.002984285,0.003728187,0.00007178078,0.00008593273,0.00003293265,0.00004662203,0.0003692302,0.0002796976,0.9910063,0.00006223263,0.001221491,0.0001112777],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9866901,0.003781093,0.007706496,0.001021341,0.00009425706,0.0004039284,0.00004450922,0.00000165691,0.0002566046],"genre_scores_gemma":[0.9950974,0.000844589,0.002470691,0.0002013789,0.001039617,0.00002198861,0.00006724038,0.00003143737,0.000225665],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01371959,"threshold_uncertainty_score":0.5297533,"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."}}