Inherited hemoglobin diseases, like sickle cell disease and thalassemias burden the life of tens of millions and cause the death of approximately 100.000 patients every year. Most of the patients are children under five in developing countries.
In this project we study the genetic modification of blood precursors in order to produce a healthy hemoglobin. We use the genetic tool CRISPR-Cas9 to modify the patient cells in a way that they would produce a source to permanently cure the diseases. Although the project aims at developing a genetic therapy, the study does not yet include therapeutic inventions. However, the hypothesis is that our results in cell cultures and animal models would produce a starting point for safety and efficacy studies.