{"id":"W4319345548","doi":"10.1016/j.cell.2023.01.005","title":"PIKFYVE inhibition mitigates disease in models of diverse forms of ALS","year":2023,"lang":"de","type":"article","venue":"Cell","topic":"Amyotrophic Lateral Sclerosis Research","field":"Medicine","cited_by":98,"is_retracted":false,"has_abstract":false,"ca_institutions":"Centre Hospitalier de l’Université de Montréal","funders":"National Institute of Neurological Disorders and Stroke; National Institute on Deafness and Other Communication Disorders; NIH Office of the Director; National Heart, Lung, and Blood Institute; Tau Consortium; Association for Frontotemporal Degeneration; National Institutes of Health; Target ALS; Discovery Institute; Bruno and Ilse Frick Foundation for Research on ALS; Harrington Discovery Institute, University Hospitals; John Douglas French Alzheimer's Foundation; University of Washington; Donald E. and Delia B. Baxter Foundation; National Human Genome Research Institute; University of Southern California; New York Stem Cell Foundation; Alzheimer's Drug Discovery Foundation; Muscular Dystrophy Association; Southern California Clinical and Translational Science Institute; California Institute for Regenerative Medicine; Milken Family Foundation; Ford Foundation; National Institute on Aging; Judith and Jean Pape Adams Charitable Foundation; U.S. Department of Defense","keywords":"Amyotrophic lateral sclerosis; Biology; TARDBP; SOD1; Disease; Mechanism (biology); C9orf72; Neuroscience; Autophagy; Protein aggregation; Ubiquitin; Genetics; Gene; Apoptosis; Medicine; Pathology","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":[0.000348133,0.0007750627,0.0009269768,0.0006256435,0.0004633837,0.0007160982,0.0005912146,0.0009400107,0.003627382],"category_scores_gemma":[0.0002707237,0.0002055677,0.0006014365,0.0003203945,0.0008726161,0.0006821605,0.0003939771,0.002495285,0.0008417688],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005988397,"about_ca_system_score_gemma":0.0008591699,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001427915,"about_ca_topic_score_gemma":0.004313449,"domain_scores_codex":[0.9996549,0.00003428398,0.00003453304,0.00005589343,0.00007761388,0.0001427553],"domain_scores_gemma":[0.9997961,0.00001631398,0.00004016937,0.00001873533,0.00002293676,0.0001057993],"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":[0.004699579,0.0007077543,0.0005500722,0.000364043,0.0001219015,0.0002096253,0.000107232,0.0006463081,0.9781068,0.0009894121,0.0020963,0.01140102],"study_design_scores_gemma":[0.0008062008,0.006786704,0.01066691,0.0001213963,0.0002536279,0.0008258263,0.000568442,0.003195576,0.9570126,0.001070815,0.01863007,0.00006179538],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9840972,0.003468132,0.002549515,0.001098577,0.0005558071,0.00009993598,0.002293187,0.0007027946,0.00513486],"genre_scores_gemma":[0.9876553,0.002173874,0.001327117,0.0002559389,0.00005248299,0.0001388486,0.001491589,0.00006394442,0.006840834],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003627382,"threshold_uncertainty_score":0.01213485,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04474635295060772,"score_gpt":0.2901526269626132,"score_spread":0.2454062740120055,"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."}}