St. Jude Reference #SJ-16-0040
Recent progress in the field of gene therapy indicates that patients afflicted with hemoglobinopathies such as beta thalassemia or sickle cell anemia will benefit from novel therapeutic hematopoietic cells (HCs) using lentiviral vectors carrying the beta globin gene, as mouse hemoglobin disorder models indicate a long term correction. Benefits include avoiding the risk of Graft Versus Host Disease (GVHD), immunosuppressive pre-transplant conditioning, and long searches for or the lack of compatible donors.
Researchers at St. Jude have developed a DNA construct containing an erythroid lineage-specific promoter from a glycophorin A gene; and a nucleotide coding sequence operably linked to the promoter sequence. Embodiments include an erythroid-specific promoter, a nucleotide coding sequence that encodes RNA or proteins (an artificial zinc finger protein), a gene therapy (viral) vector, and a method for preventing or treating a hematopoietic disorder by administering the DNA construct to a subject in need of treatment for blood diseases.
Gene therapy, hemoglobinopathies, beta thalassemia, sickle cell anemia, lentiviral vector, beta globin gene, hematopoietic disorders, erythroid.
Granted patents or published applications
Pending US application published as US 2019/0316154 A1
Related scientific references
Scott A. Peslak, Selami Demirci, Vemika Chandra, Wei Tong, John F. Tisdale, Gerd A. Blobel, et al., “Forced enhancer-promoter rewiring to alter gene expression in animal models.” Cell, Published:January 30, 2023
Naoya Uchida, Francesca Ferrara, Claire M. Drysdale, Morgan Yapundich, Jackson Gamer, Tina Nassehi, Julia DiNicola, Yoshitaka Shibata, Matthew Wielgosz, Yoon-Sang Kim, Matthew Bauler, Robert E. Throm, Juan J. Haro-Mora, Selami Demirci, Aylin C. Bonifacino, Allen E. Krouse, N. Seth Linde, Robert E. Donahue, Byoung Ryu, John F. Tisdale; “Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor,” Sci. Transl. Med., 13 (591), eabb0411. • DOI: 10.1126/scitranslmed
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