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Evans Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008884","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100001368","name":"V Foundation for Cancer Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100001368","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2026,10]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>Gene therapy is revolutionizing treatment paradigms for inherited haematological and immunological conditions. Recent successes, including the United States\u00a0Food and Drug Administration (FDA) approval of gene therapy products for sickle cell disease and beta\u2010thalassaemia, highlight the translational path of gene therapies for erythroid\u2010specific disorders. In contrast, gene therapy development for other inherited erythroid disorders remains largely preclinical. Here, we examine the emerging landscape of gene therapies for inherited non\u2010haemoglobinopathy erythroid disorders, focusing on the status of gene therapies for Diamond\u2013Blackfan anaemia (DBA), pyruvate kinase deficiency (PKD), X\u2010linked sideroblastic anaemia (XLSA), congenital erythropoietic porphyria (CEP) and congenital dyserythropoietic anaemia (CDA). We discuss the latest cellular engineering approaches being applied to developing therapies for these erythroid disorders and evolving strategies for conditioning and engraftment of modified cells. Despite the rarity of these disorders individually, several convergent biological and translational themes have emerged. Leveraging shared insights across diseases may accelerate clinical translation and broaden the curative potential of gene therapy for inherited erythroid disorders beyond the haemoglobinopathies.<\/jats:p>","DOI":"10.1111\/bjh.70675","type":"journal-article","created":{"date-parts":[[2026,7,14]],"date-time":"2026-07-14T23:58:40Z","timestamp":1784073520000},"page":"1469-1481","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies"],"prefix":"10.1111","volume":"209","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8457-329X","authenticated-orcid":false,"given":"Gaurav","family":"Joshi","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\/Oncology University of Texas Southwestern Medical Center  Dallas Texas USA"},{"name":"Department of Pediatrics Children's Medical Center  Dallas Texas USA"},{"name":"Harold C. 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