{"id":"W4412676182","doi":"10.1080/23995270.2025.2535273","title":"A plain language summary of the phase 1/2 study of AAV5-hRKp.RPGR (botaretigene sparoparvovec) in people with RPGR-associated X-linked retinitis pigmentosa","year":2025,"lang":"en","type":"article","venue":"Future Rare Diseases","topic":"Retinoids in leukemia and cellular processes","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Kellogg's (Canada)","funders":"Johnson and Johnson; Moorfields Eye Hospital NHS Foundation Trust; National Institute for Health and Care Research","keywords":"Retinitis pigmentosa; Phase (matter); Medicine; Physics; Ophthalmology","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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001133641,0.0002296162,0.0003542552,0.00007184571,0.00007594471,0.00001527512,0.0003972915,0.0001445438,0.00003336374],"category_scores_gemma":[0.0002119938,0.0001653895,0.0001196539,0.0005033865,0.0001038948,0.000006377465,0.0001601839,0.0001358489,3.466905e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003665727,"about_ca_system_score_gemma":0.0003741734,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001553503,"about_ca_topic_score_gemma":0.0009606266,"domain_scores_codex":[0.9985111,0.0002357925,0.0003687996,0.0003778934,0.000267379,0.000239004],"domain_scores_gemma":[0.9989788,0.00004267561,0.000237013,0.0005474348,0.0001348298,0.00005923692],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.001949424,0.004871637,0.8540499,0.0005806972,0.0005157356,0.00008775006,0.002230509,0.0001224145,0.1296089,0.00004481961,0.003923606,0.002014553],"study_design_scores_gemma":[0.01763579,0.003064955,0.543299,0.001112288,0.000798289,0.000009641984,0.03965623,0.00006002024,0.3899168,0.00007169745,0.003492433,0.0008827914],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9922622,0.005978373,0.00002125468,0.0000907913,0.000157568,0.0006056519,0.0003438341,0.00001471375,0.0005255766],"genre_scores_gemma":[0.9984704,0.000264915,0.00002232911,0.0001040328,0.0001170192,0.00007254088,0.0004725801,0.0000220318,0.0004541667],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.3107509,"threshold_uncertainty_score":0.674439,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.003337129755909234,"score_gpt":0.2396379515389264,"score_spread":0.2363008217830172,"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."}}