Therapeutic retroviral vectors for gene therapy
Assigneesbluebird bio, Inc., Massachusetts Institute of Technology
InventorsPhilippe Leboulch, Robert Pawliuk, Karen Westerman
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Abstract
The invention provides improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. The invention further provides improved gene therapy methods for treating hematopoietic-related disorders. Retroviral gene therapy vectors that are optimized for erythroid specific expression and treatment of hemoglobinopathic conditions are disclosed.
Claims18
A self-inactivating (SIN)lentiviral vector comprising:
- a) a modified 5′ long terminal in (LTR), wherein the promoter of the modified 5′ LTR is replaced with a heterologous promoter;
- b) an RNA export element;
- c) a lentiviral central polyurine tract or DNA FLAP (cPPT/FLAP);
- d) a β-globin promoter;
- e) a β-globin locus control region (LCR), wherein the β-globin promoter and β-globin LCR are operatively linked to a gene of interest;
- and
- f) a modified 3′ LTR, wherein the modified 3′ LTR comprises at least one deletion compared to the wild-type 3′ LTR.
The vector of claimed 1, wherein the modified 5′ LTR further comprises a deletion compared to the wild-type 5′ LTR.
Dependencies are inferred from the claim text.