{"id":"W4400189917","doi":"10.1182/blood.2023023349","title":"Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy","year":2024,"lang":"en","type":"article","venue":"Blood","topic":"Iron Metabolism and Disorders","field":"Medicine","cited_by":7,"is_retracted":false,"has_abstract":true,"ca_institutions":"Acuitas Therapeutics (Canada)","funders":"National Institute of Diabetes and Digestive and Kidney Diseases; National Heart, Lung, and Blood Institute; University of Pennsylvania; National Cancer Institute; National Institutes of Health; Pennsylvania Department of Health; National and Kapodistrian University of Athens; Eunice Kennedy Shriver National Institute of Child Health and Human Development; Children's Hospital of Philadelphia","keywords":"Genetic enhancement; Haematopoiesis; CD34; Progenitor cell; Biology; Stem cell; Viral vector; Bone marrow; Thalassemia; Transplantation; Ex vivo; Erythropoiesis; Severe combined immunodeficiency; Cancer research; Immunology; Molecular biology; Medicine; In vivo; Gene; Anemia; Internal medicine; Cell biology; Recombinant DNA; Genetics","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":[0.0004328967,0.001009116,0.0004763083,0.0008956757,0.0003235916,0.0006139711,0.0005546084,0.001083427,0.00250885],"category_scores_gemma":[0.0001363846,0.0002840707,0.0005132473,0.0003373107,0.0005881823,0.0003682037,0.000312373,0.001835787,0.001094224],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005026826,"about_ca_system_score_gemma":0.0003691322,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005689467,"about_ca_topic_score_gemma":0.0006059654,"domain_scores_codex":[0.9996079,0.00004669138,0.00005455643,0.0001007075,0.0001264357,0.00006377727],"domain_scores_gemma":[0.9997481,0.00002694735,0.00008019391,0.00003219432,0.00002922173,0.00008344463],"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":[0.0001148051,0.0002063009,0.0001242452,0.00005531215,0.00001156914,0.0001009333,0.00004040172,0.0001416212,0.9965765,0.0005206421,0.0001661181,0.001941706],"study_design_scores_gemma":[0.0001002641,0.001118408,0.0007403431,0.00002392676,0.00004456456,0.000472025,0.00002276326,0.001298009,0.9882339,0.00009610067,0.007840027,0.000009612078],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9492068,0.002648107,0.03417736,0.0006908813,0.0004442878,0.001132928,0.003354584,0.001545417,0.006799685],"genre_scores_gemma":[0.945868,0.002567292,0.02444559,0.0003850292,0.00008559166,0.001268428,0.003261352,0.0002823379,0.02183646],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00250885,"threshold_uncertainty_score":0.00839293,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03971835011419329,"score_gpt":0.2670252972844952,"score_spread":0.2273069471703019,"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."}}