St. Jude Reference #SJ-20-0020
Description
Researchers at St. Jude recreated the naturally occurring point mutation in the human γ-globin gene promoter at the position -175 (T-C) in erythroid progenitor cell line (HUDEP-2) and in adult hematopoietic stem cells (HSCs) to induced therapeutic levels of fetal globin in differentiated erythroid cells. This invention can be used to manipulate the HSCs from sickle cell disease or β-thalassemia patients to induce fetal hemoglobin which ameliorates the disease severity.
Keywords
human γ-globin gene promoter, position -175 (T-C), erythroid progenitor cell line (HUDEP-2), hematopoietic stem cells (HSCs), fetal globin, differentiated erythroid cell, sickle cell disease, β-thalassemia.
Granted patents or published applications
Pending US patent.
Related scientific references
Mayuranathan, T., Newby, G.A., Feng, R. et al. Potent and uniform fetal hemoglobin induction via base editing. Nat Genet 55, 1210–1220 (2023). https://doi.org/10.1038/s41588-023-01434-7
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