Compositions and methods for treating beta-hemoglobinopathies
AssigneesCalimmune, Inc., Calimmune Australia Pty Ltd
InventorsJeffrey AHLERS, Jeffrey BARTLETT, Chi-Lin LEE, Gene-Errol Eugenio RINGPIS, Geoffrey Phillip SYMONDS, Ming YAN
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Abstract
The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.
Claims20
A vector comprising a first expression control sequence operably linked to a first nucleic acid sequence, the first nucleic acid sequence encoding a shRNA to knockdown HPRT;
- and a second expression control sequence operably linked to a second nucleic acid sequence, the second nucleic acid sequence encoding a gamma-globin gene.
A host cell which is HPRT deficient and which expresses a peptide having SEQ ID NO:
- 4, wherein the host cell is prepared by transducing an HSC with a vector comprising a first expression control sequence operably linked to a first nucleic acid sequence, the first nucleic acid sequence encoding a shRNA to knockdown HPRT;
- and a second expression control sequence operably linked to a second nucleic acid sequence, the second nucleic acid sequence encoding a gamma-globin gene.
A host cell comprising:
- (i) at least one of a nucleic acid molecule having SEQ ID NO:
- 3 or a peptide having SEQ ID NO:
- 4;
- and (ii) at least one of a nucleic acid molecule having SEQ ID NO:
- 1 or a nucleic acid molecule having SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.