{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.000474219,0.0008871395,0.0005388293,0.001036541,0.0003532256,0.0006817323,0.0007869281,0.001090006,0.001387274],"category_scores_gemma":[0.0002070434,0.0004548652,0.0004445189,0.0002966801,0.0006305971,0.0005851118,0.0003601223,0.001788944,0.0005086343],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004530924,"about_ca_system_score_gemma":0.0006088432,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007618371,"about_ca_topic_score_gemma":0.0007769335,"domain_scores_codex":[0.9996392,0.00005090487,0.00004919173,0.00008506543,0.0001039206,0.00007168174],"domain_scores_gemma":[0.9997296,0.00003320156,0.00007447213,0.00004093813,0.00002642696,0.00009535791],"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":[0.0001317937,0.0002208947,0.00009209701,0.00004485737,0.000009741191,0.0001279162,0.00003629146,0.000202105,0.9959507,0.0006641722,0.0001138005,0.002405658],"study_design_scores_gemma":[0.000223884,0.002057179,0.001130424,0.00003950954,0.0000641305,0.000958839,0.00003312278,0.00306744,0.9830266,0.0002825569,0.009097814,0.00001845411],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9433723,0.003662576,0.04398632,0.0006660244,0.0003145623,0.0008180568,0.001757026,0.001135023,0.004288006],"genre_scores_gemma":[0.9425438,0.003997868,0.03563309,0.0002450881,0.00007932964,0.001224568,0.002398976,0.0002387836,0.01363846],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001387274,"threshold_uncertainty_score":0.004640877,"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."}}