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US 2019/0316154 A1Published application
Erythroid-specific promoter and method of use thereof
AssigneeSt. Jude Children's Research Hospital
InventorsByoung Ryu, Matthew M. Wielgosz, Francesca Ferrara
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Abstract
A DNA construct containing an erythroid lineage-specific promoter operably linked to a nucleotide coding sequence of interest and a method of using the same in the prevention or treatment of a hematopoietic disorder such as a hemoglobinopathy are described. Further disclosed are erythroid-specific promoters and erythroid-specific enhancers that can be used in the DNA construct.
Claims17
A DNA construct comprising:
- an erythroid lineage-specific promoter from a glycophorin A gene;
- and
- a nucleotide coding sequence operably linked to the promoter sequence.
A DNA construct comprising:
- an erythroid lineage-specific glycophorin A, Ankyrin, beta-Spectrin or Adducine 2 promoter;
- a BCL11A+58, GATA-1 or HS40 enhancer;
- and
- a nucleotide sequence encoding a BCL11A inhibitory RNA molecule operably linked to the promoter.
1 more element
Dependencies are inferred from the claim text.