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US 5,861,488 AGranted
Compositions and methods for treating sickle cell disease
AssigneeMassachusetts Institute of Technology
InventorsPhilippe LeBoulch, Irving M. London
Life of this patent
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Abstract
Gene therapy methods and compositions for high level expression of anti-sickling globin proteins in erythroid cells for treating Sickle Cell disease are described.
Claims11
An anti-sickling .beta.-globin protein having structure of human .beta.-globin and comprising the amino acid sequence shown in SEQ ID NO:
- 1, wherein at least one amino acid residue of SEQ ID NO:
- 1 has been substituted with an anti-sickling amino acid residue selected from the group consisting of:
- 9 Thr, 12 Asn, 22 Ala, 50 Ser, 80 Asp, 86 Ser, 87 Gln, 116 Arg, 117 Asn, 125 Gln and 126 Met, provided that said anti-sickling .beta.-globin protein does not contain all of said anti-sickling residues, wherein said anti-sickling .beta.-globin protein exhibits reduced sickling compared to wild-type .beta.-globin protein when present in a hemoglobin tetramer along with at least one .beta. 6 Val mutated .beta.-globin protein.
Dependencies are inferred from the claim text.