Globin gene therapy for treating hemoglobinopathies
AssigneeMemorial Sloan Kettering Cancer Center
InventorsMichel Sadelain, Annalisa Cabriolu
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Abstract
The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.
Claims19
A method of treating a hemoglobinopathy in a subject, comprising administering an effective amount of an autologous cell transduced with an expression cassette to the subject, wherein the expression cassette comprises:
- a) a globin gene or a functional portion thereof operably linked to a β-globin locus control region (LCR) that comprises a Dnase I hypersensitive site-2 (HS2) region, a Dnase I hypersensitive site-3 (HS3) region, and a Dnase I hypersensitive site-4 (HS4) region, wherein the HS4 region has a length of between 600 bp and 900 bp and does not comprise the nucleotide sequence set forth in SEQ ID NO:
- 50;
- or
- b) a globin gene or a functional portion thereof operably linked to a β-globin locus control region (LCR) that comprises a HS1 region, a HS3 region, and a HS4 region and does not comprise a core sequence of HS2 region, wherein the HS4 region has a length of between 600 bp and 900 bp does not comprise the nucleotide sequence set forth in SEQ ID NO:50.
Dependencies are inferred from the claim text.