{"id":"W2613512540","doi":"10.1016/j.omtm.2017.05.003","title":"Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34 + Cells for Correction of Fabry Disease","year":2017,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":46,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of British Columbia; University of Guelph; Western University; Juravinski Hospital; Université de Sherbrooke; Calgary Laboratory Services; Dalhousie University; University Health Network; University of Toronto; Alberta Children's Hospital; University of Calgary","funders":"Canadian Institutes of Health Research; Cumming School of Medicine, University of Calgary; Kidney Foundation of Canada; University of Calgary; Alberta Health Services","keywords":"CD34; Fabry disease; Haematopoiesis; Genetic enhancement; Hematopoietic stem cell transplantation; Xenotransplantation; Viral vector; Stem cell; Transplantation; Ex vivo; Immunology; Cancer research; Medicine; Recombinant DNA; Biology; Internal medicine; In vivo; Disease; Cell biology; Biochemistry","routes":{"ca_aff":true,"ca_fund":true,"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":[0.001768592,0.0006814584,0.0004449982,0.000456937,0.0002792575,0.0008532034,0.0006316915,0.000826421,0.001351683],"category_scores_gemma":[0.0009747206,0.0002246099,0.0004298814,0.0001986201,0.0004657502,0.0003049916,0.0003854526,0.001149874,0.0005448369],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006208597,"about_ca_system_score_gemma":0.0005599852,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009988976,"about_ca_topic_score_gemma":0.001166695,"domain_scores_codex":[0.9988991,0.0002873315,0.0001359847,0.0001468036,0.0003993519,0.000131368],"domain_scores_gemma":[0.9995018,0.0001242342,0.00009282953,0.00008477549,0.0001267349,0.00006953876],"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":[0.0001121028,0.0003628951,0.0004066167,0.00006803284,0.00001520622,0.000105176,0.00006585051,0.0004685792,0.9915114,0.000211751,0.0003089722,0.006363452],"study_design_scores_gemma":[0.00003854085,0.001937126,0.001157433,0.00001159565,0.00003529661,0.0002288016,0.00001694755,0.001553692,0.9911349,0.00004650935,0.003828814,0.0000103861],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9009208,0.001120181,0.08544965,0.0004076614,0.0001660913,0.004301644,0.002174512,0.001096685,0.004362586],"genre_scores_gemma":[0.8901302,0.001299473,0.09544948,0.0002755629,0.00003731405,0.002187893,0.0038355,0.0002378931,0.006546647],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001768592,"threshold_uncertainty_score":0.00935328,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1693160542515036,"score_gpt":0.5115581452344975,"score_spread":0.3422420909829939,"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."}}