首页>
外国专利>
Recombinant lentivector encoding the human globin gene, its use in the treatment of hemoglobinopathies, mammalian cells derived from the hematopoietic progenitor, and method for preparing a therapeutic composition for treating hemoglobinopathy in a mammal
Recombinant lentivector encoding the human globin gene, its use in the treatment of hemoglobinopathies, mammalian cells derived from the hematopoietic progenitor, and method for preparing a therapeutic composition for treating hemoglobinopathy in a mammal
"RECOMBINANT LENTIVETOR CODIFYING THE HUMAN GLOBINE GENE, USE OF THE SAME IN THE TREATMENT OF HEMOGLOBINOPATHIES, MAMMALIUM CELLS FROM THE PROGENITOR, HEMATOPOIETIC AND METHOD TO PREPARE A COMPOSIUM THERAPYOPHEMA Recombinant lentiviral vectors having a region encoding a functional globin gene; and large parts of the beta-globulin locus control regions that include HSase, HS3 and HS4 Hypersensitive Dnase I Sites provide for the expression of beta-globin when introduced into a mammal, for example a human, in vivo. Optionally, the vector further includes a region encoding a dihydrofolate reductase. The vector may be used to treat hemoglobinopathies, including beta-thalassemia and sickle cell anemia. For example, hematopoietic progenitor cells or stem cells may be transformed ex vivo and then returned to the patient. Selection processes can be used to increase the percentage of transformed cells in the returned population. For example, a selection marker that makes transformed cells more drug resistant than non-transformed cells allows selection by treating cells with the corresponding drug.
展开▼
