{"id":"W4400255086","doi":"10.20944/preprints202406.2068.v1","title":"Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges","year":2024,"lang":"en","type":"preprint","venue":"Preprints.org","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":5,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"","keywords":"Spinal muscular atrophy; SMN1; SMA*; Genetic enhancement; Motor neuron; Antisense therapy; Medicine; Gene; Neuroscience; Bioinformatics; Biology; Oligonucleotide; Spinal cord; Genetics; Computer science","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":[0.004620053,0.0007869862,0.001125012,0.0009312899,0.0003579637,0.002238677,0.000726313,0.001446473,0.006343485],"category_scores_gemma":[0.002607423,0.000237481,0.0006457203,0.0008539383,0.00135914,0.002539889,0.00094099,0.002926718,0.001725028],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0008991619,"about_ca_system_score_gemma":0.001395703,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005384134,"about_ca_topic_score_gemma":0.0008423927,"domain_scores_codex":[0.999353,0.0001737334,0.00004868952,0.0001168114,0.0002196389,0.00008811119],"domain_scores_gemma":[0.9975716,0.001623675,0.0001560731,0.0001130499,0.0003538422,0.0001818495],"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":[0.001564448,0.0006303716,0.001252515,0.006227662,0.0002122808,0.0004145231,0.0002811947,0.002899546,0.04945287,0.0414135,0.02202608,0.873625],"study_design_scores_gemma":[0.0003285676,0.00352732,0.00304191,0.001721762,0.0004171805,0.00195628,0.0003899587,0.003109833,0.045669,0.03263723,0.9071193,0.00008157694],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0103886,0.9496128,0.01384292,0.01343723,0.0009182441,0.00005488912,0.0001667027,0.0002403371,0.01133827],"genre_scores_gemma":[0.05153332,0.9255062,0.01230517,0.003357667,0.001808935,0.0001230564,0.0004271939,0.00008411123,0.004854307],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.006343485,"threshold_uncertainty_score":0.02443349,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1968572707964944,"score_gpt":0.4069118120994002,"score_spread":0.2100545413029059,"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."}}