Compositions and methods for hemoglobin production
AssigneeThe Children's Hospital of Philadelphia
InventorsStefano Rivella, Laura Breda, Alisa Dong, Silvia Pires Lourenco, Amaliris Gonzalez
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Abstract
In one aspect of the instant invention, lentiviral vectors are provided, particularly for treating hemoglobinopathies. Composition comprising the lentiviral vector are also encompassed by the instant invention. In accordance with another aspect of the instant invention, methods of inhibiting, treating, and/or preventing a hemoglobinopathy (e.g., sickle cell disease or thalassemia) in a subject are provided. In a particular embodiment, the method comprises administering a viral vector of the instant invention to a subject in need thereof hemoglobinopathy. In a particular embodiment, the subject has sickle cell anemia.
Claims10
A lentiviral vector comprising a nucleic acid molecule comprising:
- i) a 5′ long terminal repeat (LTR) and a 3′ LTR, wherein one of said LTRs is self-inactivating;
- ii) at least one polyadenylation signal;
- iii) at least one promoter;
- iv) a globin gene locus control region (LCR), wherein said LCR comprises HS1, HS2, HS3, and HS4, wherein said LCR comprises the nucleotide sequence of nucleotides 6766-10641 of SEQ ID NO:
- 1;
- v) an ankyrin insulator element (Ank);
- vi) a Woodchuck Post-Regulatory Element (WPRE), wherein the WPRE is 3′ of the 3′LTR;
- and
- vii) a sequence encoding human beta-globin.
A lentiviral vector comprising SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.