In vitro mass production of human erythroid cells from the blood of normal donors and thalassemic patients .

Two-phase culture method that allows mass production of primary human erythroid cells, starting from the light-density cells of normal donors and thalassemic patients.

Summary of Invention The present invention relates to a new two-phase culture method that allows mass production of primary human erythroid cells, starting from the light-density cells of normal donors and thalassemic patients. In both cases, highly homogeneous primary erythroid cells are produced in numbers sufficient for biochemical and molecular studies from very modest volumes (10 mL) of blood. Therefore, this method will be useful for donor-specific studies of hemoglobin F reactivation, to identify inducers targeted for each specific patient. Solution to which problem (s) The present invention allows to obtain In Vitro Mass Production of Human Erythroid Cells from the Blood of Normal Donors and of Thalassemic Patients. Most of the biochemical studies on the characterization of erythroid cells, which require a mass number of cells, were and still are performed using cell lines as a model. Immortalized cell lines are not a perfect mirror of primary cells, as many regulatory pathways have been altered during the transformation process that led to their immortalization. In vitro unilineage primary erythroid cells are a good model, and can be obtained from neonatal blood or from adult marrow and cultured in the presence of stimulators of the glucocorticoid receptor, such as dexamethasone. The present invention proposes a valid solution to this problem. Key applications - In vitro testing for safety and efficacy of gene therapy for mediterraneam anemia and beta-thalassemia. The type and the amount of produced hemoglobin will be a measure of the efficacy of the therapy, while the number of the integrated sites, their localization and the transcription of known oncogenes will be a measure of the safety - In vitro screening of new drug agents to treat mediterraneam anemia and beta-thalassemia. In vitro screening of patients responding to fetal hemoglobin induction therapy, such as with some chemo-therapeutic agents and hidroxyurea. Stage of Development The method is fully developed and scaleable.

Phase I

Priority Filing in US, Feb.27, 2003

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