{"doi":"10.3390/cells11111843","title":"Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease","abstract":"Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is caused by a point mutation (20A > T) in the β-globin gene. Since SCD is the most common single-gene disorder, curing SCD is a primary goal in HSC gene therapy. β-thalassemia results from either the absence or the reduction of β-globin expression, and it can be cured using similar strategies. In HSC gene-addition therapy, patient CD34+ HSCs are genetically modified by adding a therapeutic β-globin gene with lentiviral transduction, followed by autologous transplantation. Alternatively, novel gene-editing therapies allow for the correction of the mutated β-globin gene, instead of addition. Furthermore, these diseases can be cured by γ-globin induction based on gene addition/editing in HSCs. In this review, we discuss HSC-targeted gene therapy in SCD with gene addition as well as gene editing.","journal":"Cells","year":2022,"id":238873,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":52,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9554,"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":662660,"name":"Malikiya Hinds","orcid":null,"position":1,"is_corresponding":false},{"id":792048,"name":"Anh‐Tuan Le","orcid":"0000-0002-5648-3414","position":2,"is_corresponding":false},{"id":863427,"name":"Rebecca Chu","orcid":"0000-0002-6750-1155","position":3,"is_corresponding":false},{"id":863428,"name":"Xiong Liu","orcid":"0000-0001-9021-3031","position":4,"is_corresponding":false},{"id":320679,"name":"Naoya Uchida","orcid":"0000-0001-6322-8991","position":5,"is_corresponding":false},{"id":864081,"name":"Paula Germino-Watnick","orcid":null,"position":0,"is_corresponding":true}],"reference_count":232,"raw_metadata":null,"created_at":"2026-07-19T00:22:31.741535Z","pmid":"35681538","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":[]}