{"doi":"10.1016/j.isci.2022.104374","title":"High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation","abstract":"Background: A point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin subunit of hemoglobin, with resultant anemia and multiorgan damage that typically shortens lifespan by decades. Because SCD is caused by a single mutation, and hematopoietic stem cells (HSCs) can be harvested, manipulated, and returned to an individual, it is an attractive target for gene correction. Results: erythroid differentiation markedly enriches for corrected β-globin alleles, indicating that erythroblasts carrying one or more corrected alleles have a survival advantage. Significance: These findings indicate that the sickle mutation can be corrected in autologous HSCs with an optimized protocol suitable for clinical translation.","journal":"iScience","year":2022,"id":245978,"datarank":1.4629302032670033,"base_score":3.970291913552122,"endowment":3.970291913552122,"self_citation_contribution":0.5955437870328184,"citation_network_contribution":0.8673864162341848,"self_endowment_contribution":0.5955437870328184,"citer_contribution":0.8673864162341848,"corpus_percentile":null,"corpus_rank":null,"citation_count":52,"citer_count":45,"citers_with_citation_signal":38,"citers_with_endowment":38,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.954,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2022-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":419791,"name":"Mark A. DeWitt","orcid":"0000-0001-8784-0026","position":1,"is_corresponding":false},{"id":273395,"name":"Stacia K. Wyman","orcid":"0000-0002-8937-8397","position":2,"is_corresponding":false},{"id":327004,"name":"Jonathan T. Vu","orcid":"0000-0002-4950-7967","position":3,"is_corresponding":false},{"id":823453,"name":"Seok‐Jin Heo","orcid":"0009-0004-4830-8319","position":4,"is_corresponding":false},{"id":497695,"name":"Shirley Shao","orcid":"0000-0003-0326-9083","position":5,"is_corresponding":false},{"id":881626,"name":"Finn Hennig","orcid":null,"position":6,"is_corresponding":false},{"id":463220,"name":"Zulema Romero","orcid":"0000-0001-8305-8184","position":7,"is_corresponding":false},{"id":881627,"name":"Beatriz Campo-Fernández","orcid":null,"position":8,"is_corresponding":false},{"id":823724,"name":"Suzanne Saïd","orcid":null,"position":9,"is_corresponding":false},{"id":18369,"name":"Matthew S. McNeill","orcid":"0000-0002-8737-785X","position":10,"is_corresponding":false},{"id":18368,"name":"Garrett R. Rettig","orcid":"0000-0003-4634-5787","position":11,"is_corresponding":false},{"id":823454,"name":"Yongming Sun","orcid":"0000-0002-3398-4267","position":12,"is_corresponding":false},{"id":610888,"name":"Yu Wang","orcid":"0000-0001-7811-5834","position":13,"is_corresponding":false},{"id":18371,"name":"Mark A. Behlke","orcid":"0000-0002-4218-857X","position":14,"is_corresponding":false},{"id":3656,"name":"Donald B. Kohn","orcid":"0000-0003-1840-6087","position":15,"is_corresponding":false},{"id":316930,"name":"Dario Boffelli","orcid":"0000-0003-2529-9056","position":16,"is_corresponding":false},{"id":632130,"name":"Mark C. Walters","orcid":"0000-0002-6515-4559","position":17,"is_corresponding":false},{"id":233291,"name":"Jacob E. Corn","orcid":"0000-0002-7798-5309","position":18,"is_corresponding":false},{"id":318974,"name":"David I. K. Martin","orcid":null,"position":19,"is_corresponding":false},{"id":823722,"name":"Wendy Magis","orcid":null,"position":0,"is_corresponding":true}],"reference_count":55,"raw_metadata":{"citation_network_status":"fetched"},"created_at":"2026-07-19T00:23:39.284121Z","pmid":"35633935","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}