{"id":"W2067074806","doi":"10.1038/gt.2012.43","title":"Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial","year":2012,"lang":"en","type":"article","venue":"Gene Therapy","topic":"Lipid metabolism and disorders","field":"Medicine","cited_by":399,"is_retracted":false,"has_abstract":false,"ca_institutions":"Université de Montréal; Cégep de Chicoutimi; Université du Québec à Chicoutimi","funders":"Canadian Institutes of Health Research; Health Canada; Canada Research Chairs; Heart and Stroke Foundation of Canada","keywords":"Hypertriglyceridemia; Lipoprotein lipase; Pancreatitis; Medicine; Internal medicine; Gastroenterology; Genetic enhancement; Acute pancreatitis; Lipoprotein; Endocrinology; Triglyceride; Biology; Cholesterol; Adipose tissue","routes":{"ca_aff":true,"ca_fund":true,"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":[0.002397876,0.001707632,0.00332585,0.0006219634,0.000574491,0.001835803,0.0009074637,0.002940743,0.002754456],"category_scores_gemma":[0.00135638,0.0005007369,0.002366206,0.000522175,0.002384859,0.002314782,0.0003272435,0.00365328,0.001062179],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0008981974,"about_ca_system_score_gemma":0.0006518821,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003608326,"about_ca_topic_score_gemma":0.0003079902,"domain_scores_codex":[0.9987526,0.0005087503,0.0000951046,0.0002371147,0.0001657846,0.0002406581],"domain_scores_gemma":[0.9989489,0.000332265,0.000240654,0.0001604644,0.00007488632,0.0002428308],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.8976653,0.02714517,0.001209752,0.0004510562,0.0008888182,0.0001184793,0.0001083934,0.0006842515,0.02572167,0.0002380608,0.0006575838,0.0451114],"study_design_scores_gemma":[0.4778615,0.5007496,0.00498219,0.00006910574,0.000981679,0.0001461806,0.00008185593,0.001198992,0.01156527,0.000432341,0.001861522,0.00006970794],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9911019,0.004785124,0.001019829,0.0003286728,0.0002896499,0.0006206375,0.0004740101,0.00007657895,0.001303531],"genre_scores_gemma":[0.9915888,0.002462761,0.0007037511,0.0004449386,0.0004454139,0.001104463,0.0007802747,0.00003209297,0.002437582],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00332585,"threshold_uncertainty_score":0.01268137,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.07772679673581326,"score_gpt":0.3550713739956776,"score_spread":0.2773445772598644,"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."}}