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US 2009/0156534 A1Published application
Globin lentiviral vectors for treatment of disease
AssigneeMemorial Sloan Kettering Cancer Center
InventorsLeszek Lisowski, Michel Sadelain
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Abstract
The invention provides compositions and methods for the treatment or prevention of disease, including, for example, β-thalassemia, anemias (e.g., sickle cell anemia) and other hemoglobinopathologies.
Claims46
A recombinant vector comprising:
- (a) a nucleotide sequence encoding a heterologous protein;
- (b) a nucleotide sequence encoding an enhancer of a β-globin locus control region (LCR) which consists of operably joined Dnase1 hypersensitive site (HS) spanning-fragments;
- and
- (c) a nucleotide sequence encoding an HS1 fragment consisting of about nucleotides 20900 to 21973 of the human β-globin LCR sequence of GenBank Accession No. NG—000007.3 (NG7) or the corresponding sequences from a mammalian β-globin LCR;
- said vector providing expression of said protein when introduced into a mammal in vivo.
2 more elements
Dependencies are inferred from the claim text.