{"id":"W4403611477","doi":"10.3390/genes15101342","title":"Recent Progress of Antisense Oligonucleotide Therapy for Superoxide-Dismutase-1-Mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen","year":2024,"lang":"en","type":"review","venue":"Genes","topic":"Amyotrophic Lateral Sclerosis Research","field":"Medicine","cited_by":12,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Faculty of Medicine and Dentistry, University of Alberta; Canadian Institutes of Health Research; Muscular Dystrophy Canada; University of Alberta; Alberta Innovates - Health Solutions; Women and Children's Health Research Institute; Children's Health Research Institute; Alberta Innovates; Heart and Stroke Foundation of Canada; U.S. Department of Defense","keywords":"Amyotrophic lateral sclerosis; Superoxide dismutase; Oligonucleotide; SOD1; Biology; Medicine; Cancer research; Virology; Genetics; Gene; Disease; Pathology; Biochemistry; Enzyme","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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0004827105,0.00097961,0.003107389,0.0006493031,0.0001183292,0.0001124701,0.0004594538,0.0006265586,0.0001936738],"category_scores_gemma":[0.00007736609,0.0006362298,0.001277177,0.0009175864,0.0003480369,0.00008143178,0.0001927438,0.0007096315,0.0003205072],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003427855,"about_ca_system_score_gemma":0.0006471287,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00004188057,"about_ca_topic_score_gemma":0.000005284919,"domain_scores_codex":[0.995182,0.0003309447,0.001360802,0.00118985,0.0009028281,0.001033611],"domain_scores_gemma":[0.997631,0.0001778255,0.0003383899,0.001059611,0.0004072495,0.0003858873],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0006054177,0.0003530368,0.00002615068,0.01337709,0.001342637,0.0001905518,0.0000821517,0.000001336891,0.0009594978,0.00005256945,0.003033487,0.9799761],"study_design_scores_gemma":[0.002671438,0.002973422,0.0004334613,0.04387828,0.002341773,0.0001653143,0.00001288433,0.00004509781,0.002147582,0.0001548099,0.9442508,0.0009251946],"study_design_candidate":"design_other","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.03021113,0.9617594,0.000001916486,0.00112871,0.0005688294,0.00545284,0.0005911253,0.0002286059,0.00005736629],"genre_scores_gemma":[0.003933131,0.9923617,0.0003362444,0.00009921179,0.0006959364,0.001024879,0.0006019132,0.000374809,0.0005721792],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9790509,"threshold_uncertainty_score":0.9996089,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1370977705371468,"score_gpt":0.3884359873114527,"score_spread":0.2513382167743059,"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."}}