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Genotype:RISPR: What Is It and How Does It Work?
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In recent years, gene therapies based on CRISPR have changed from a promising idea to a clinical reality. One of the most important milestones was the approval of the first CRISPR-based therapy in 2023, for the treatment of haematological diseases such as sickle cell anemia and beta-thalassemia. This therapy is based on the genetic editing of hematopoietic stem cells of the patient himself to restore the production of fetal hemoglobin. It is alr…
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