{"id":"W7139157448","doi":"","title":"Development of an Effective, Durable, and Safe Lentiviral Gene Therapy in a Mouse Model of Purine Nucleoside Phosphorylase Deficiency","year":2025,"lang":"","type":"dissertation","venue":"TSpace (University of Toronto)","topic":"Biochemical and Molecular Research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Toronto","funders":"","keywords":"Purine nucleoside phosphorylase; Genetic enhancement; Viral vector; Immune system; Haematopoiesis; Stem cell; Purine; Bone marrow; Ex vivo","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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0002763005,0.0003349671,0.0005963342,0.0001111242,0.0001117455,0.000006989518,0.0004568167,0.0005024428,0.0001641876],"category_scores_gemma":[0.00003139946,0.0004110776,0.0001798178,0.0001103604,0.0002414803,0.00002871742,0.0002816944,0.0001871971,3.490994e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001118247,"about_ca_system_score_gemma":0.0005175657,"about_ca_topic_candidate":true,"about_ca_topic_consensus":true,"about_ca_topic_score_codex":0.01351146,"about_ca_topic_score_gemma":0.02461117,"domain_scores_codex":[0.9982352,0.0001193302,0.000315736,0.0006605077,0.0003208944,0.0003482884],"domain_scores_gemma":[0.9987767,0.00001575908,0.0003398362,0.0004170155,0.0003155378,0.0001351452],"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":[0.00306231,0.0008151972,0.0001092699,0.0006609902,0.0001681599,0.000003963991,0.004516538,0.00009499833,0.9055432,0.00003668001,0.000005594683,0.08498308],"study_design_scores_gemma":[0.002454962,0.000722051,0.01034765,0.0001613134,0.00005139545,6.263933e-7,0.004438145,0.001663948,0.979767,0.000008755944,0.00005156471,0.0003326022],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9885117,0.007082286,0.0007271297,0.00001759799,0.0000263852,0.0007063024,0.00009179702,0.000003627553,0.002833213],"genre_scores_gemma":[0.9810849,0.007643916,0.007358761,0.000003557958,0.000004527124,0.000003157774,0.0003163578,0.00001901453,0.003565812],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.08465048,"threshold_uncertainty_score":0.9998341,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01048122059017836,"score_gpt":0.2495215849297772,"score_spread":0.2390403643395989,"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."}}