{"id":"W3176240450","doi":"10.3390/ijms22136751","title":"Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models","year":2021,"lang":"en","type":"article","venue":"International Journal of Molecular Sciences","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":11,"is_retracted":false,"has_abstract":true,"ca_institutions":"Queen's University","funders":"","keywords":"Sandhoff disease; Immune system; Genetic enhancement; Biology; HEXA; Immunocompetence; Immunology; Transgene; Hexosaminidase; Adoptive cell transfer; Gene; T cell; Genetics; Enzyme; Biochemistry","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":[0.0003894928,0.0007496236,0.0006936464,0.0006425857,0.0002497528,0.0002848817,0.0002545366,0.0008969423,0.001303108],"category_scores_gemma":[0.0001460744,0.0001832692,0.0005186746,0.0002275663,0.0004075243,0.0003875784,0.0001921839,0.001762119,0.0003032069],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002270187,"about_ca_system_score_gemma":0.0002516336,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003034475,"about_ca_topic_score_gemma":0.0003929313,"domain_scores_codex":[0.9996105,0.00005937651,0.00004080028,0.00008853793,0.00009620073,0.0001047248],"domain_scores_gemma":[0.9998291,0.00002163222,0.00004558765,0.00001741925,0.00002095316,0.00006536995],"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":[0.00103148,0.0012975,0.0003408701,0.00009354612,0.00001519843,0.00009421051,0.00006295231,0.0001277616,0.9937064,0.0001517688,0.0001237862,0.002954672],"study_design_scores_gemma":[0.0003234691,0.02169944,0.005970093,0.00002058461,0.0001070894,0.0004886697,0.00009849343,0.001725789,0.9655756,0.0001480088,0.00382062,0.00002223237],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9940845,0.001118578,0.002435242,0.0001509376,0.0001331671,0.0002186421,0.0003919744,0.0001015055,0.001365445],"genre_scores_gemma":[0.9847198,0.00157975,0.00419621,0.0002437978,0.0000714917,0.0008294769,0.0009172733,0.00003046004,0.007411775],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001303108,"threshold_uncertainty_score":0.004359305,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03710426929095083,"score_gpt":0.3419945225957688,"score_spread":0.304890253304818,"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."}}