{"id":"W2984313633","doi":"10.1182/blood-2019-126746","title":"Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study","year":2019,"lang":"en","type":"article","venue":"Blood","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":3,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Alberta","funders":"","keywords":"Busulfan; Medicine; Genetic enhancement; Severe combined immunodeficiency; Adverse effect; Bone marrow; Internal medicine; Hematopoietic stem cell transplantation; Viral vector; Gastroenterology; Transplantation; Surgery; Oncology; Immunology; Biology; In 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":[0.00202969,0.0007622031,0.0006864076,0.00026339,0.0001509951,0.000631774,0.0004529421,0.0004504802,0.0009121135],"category_scores_gemma":[0.0004912433,0.0001389024,0.0004968792,0.0001815181,0.0004657624,0.0004009255,0.0001708868,0.0008969517,0.0002056961],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003956035,"about_ca_system_score_gemma":0.0004874007,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004141513,"about_ca_topic_score_gemma":0.0004584529,"domain_scores_codex":[0.999622,0.0001670803,0.00003763842,0.00003383856,0.00008066231,0.00005871863],"domain_scores_gemma":[0.9997931,0.00005117294,0.00005294307,0.00003069424,0.00002802474,0.00004409087],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.0156447,0.01509495,0.008084604,0.0006517191,0.0005755793,0.0005292885,0.0002693199,0.001822395,0.7369338,0.0008193933,0.002641031,0.2169331],"study_design_scores_gemma":[0.01665388,0.3343272,0.03928388,0.0001629525,0.0008947469,0.002824351,0.0001708275,0.004574529,0.5802267,0.0005815448,0.0202428,0.00005662458],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9874676,0.004639104,0.004049439,0.0004068557,0.00005369689,0.0008773622,0.0006414183,0.0002070746,0.001657542],"genre_scores_gemma":[0.98498,0.004914899,0.005932549,0.0003198397,0.00006228741,0.0006838113,0.001797925,0.0000527526,0.001255968],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00202969,"threshold_uncertainty_score":0.01073414,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03686369980199877,"score_gpt":0.3412326804800742,"score_spread":0.3043689806780754,"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."}}