{"id":"W4386393174","doi":"10.1089/hum.2023.109","title":"Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders","year":2023,"lang":"en","type":"review","venue":"Human Gene Therapy","topic":"Genetic Neurodegenerative Diseases","field":"Neuroscience","cited_by":10,"is_retracted":false,"has_abstract":true,"ca_institutions":"BC Children's Hospital; University of British Columbia","funders":"Université de Lausanne; European Commission; Schweizerischer Nationalfonds zur Förderung der Wissenschaftlichen Forschung; National Science Foundation","keywords":"Huntingtin; Exon; Biology; Gene knockin; Genome editing; Huntingtin Protein; Exon skipping; CRISPR; Genetics; Trinucleotide repeat expansion; Cas9; Molecular biology; Gene; Mutant; Allele; Alternative splicing","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":[0.001964153,0.0004651383,0.0008107419,0.0004044954,0.0001613744,0.001277783,0.0006100319,0.0007142799,0.001134215],"category_scores_gemma":[0.001138584,0.0002605625,0.0004152271,0.0001576502,0.0005304559,0.0004806994,0.0005078594,0.001110307,0.0007885397],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005482091,"about_ca_system_score_gemma":0.0004200127,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003860691,"about_ca_topic_score_gemma":0.0007137181,"domain_scores_codex":[0.998673,0.000375308,0.00009655884,0.0002592708,0.0005299851,0.00006596356],"domain_scores_gemma":[0.998961,0.0004975369,0.0001823444,0.00009147871,0.0001972143,0.00007051219],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"theoretical_or_conceptual","study_design_scores_codex":[0.0003488724,0.0001559761,0.000943596,0.001235742,0.00009232745,0.000244188,0.0001588186,0.001391995,0.8758028,0.002470011,0.001382223,0.1157735],"study_design_scores_gemma":[0.00009841339,0.003803834,0.004775756,0.000406916,0.0003195014,0.005795142,0.000231222,0.01250226,0.859988,0.002899543,0.1090948,0.0000846797],"study_design_candidate":"theoretical_or_conceptual","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"review","genre_scores_codex":[0.4859797,0.2589171,0.2323171,0.006412852,0.000887174,0.0004953379,0.000886341,0.001249768,0.01285445],"genre_scores_gemma":[0.8200563,0.07941951,0.09152223,0.001754426,0.000246453,0.0004458191,0.0006260579,0.0002461728,0.00568307],"genre_candidate":"review","genre_consensus":null,"teacher_disagreement_score":0.001964153,"threshold_uncertainty_score":0.01038754,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.313917761784493,"score_gpt":0.379506074047133,"score_spread":0.06558831226264006,"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."}}