{"id":"W4405048568","doi":"10.1182/blood-2024-203983","title":"In Vitro and In Vivo Potency Differences between AAVRh74var and AAV5 Vectors Encoding the Same High-Activity Human Factor IX Variant, FIX-R338L, Expression Cassette: Implications for Hemophilia B Gene Therapy","year":2024,"lang":"en","type":"article","venue":"Blood","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Pfizer (Canada)","funders":"","keywords":"Capsid; Factor IX; Adeno-associated virus; Genetic enhancement; In vivo; Biology; Molecular biology; In vitro; Transgene; Recombinant DNA; Virology; Vector (molecular biology); Gene; Virus; Genetics","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":true,"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":[0.001365354,0.0003365298,0.000271537,0.000400374,0.0001407747,0.0008051521,0.0002516669,0.0004238494,0.001741983],"category_scores_gemma":[0.001006752,0.0002095967,0.0003114681,0.0001899163,0.000267802,0.0003916746,0.0001316706,0.0008095977,0.0005361047],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000465137,"about_ca_system_score_gemma":0.0002708089,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009802628,"about_ca_topic_score_gemma":0.0008724889,"domain_scores_codex":[0.9988241,0.000458622,0.0001409624,0.000154121,0.0002971859,0.0001250569],"domain_scores_gemma":[0.9990902,0.0004628483,0.0001786696,0.00008393019,0.0001017819,0.00008260895],"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":[0.000490289,0.0002224125,0.0008208064,0.00007601497,0.00002303393,0.00003900621,0.0000621696,0.000324173,0.9925614,0.0002537498,0.0001744596,0.004952367],"study_design_scores_gemma":[0.0000876604,0.005347038,0.009194774,0.00002519941,0.00008976941,0.0008238577,0.00007603359,0.001462041,0.9788771,0.0001744044,0.003825681,0.00001651754],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9688559,0.007813026,0.01653582,0.0002732438,0.0001342027,0.0001604273,0.000974205,0.0001252561,0.005127882],"genre_scores_gemma":[0.9923524,0.001122849,0.003837388,0.00008189323,0.00003126395,0.00007905259,0.001075191,0.00003420991,0.001385909],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001741983,"threshold_uncertainty_score":0.007220745,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03499840181854922,"score_gpt":0.3192212194955266,"score_spread":0.2842228176769773,"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."}}