Researchers sought to determine whether deferiprone would have the safe efficacy as deferoxamine for treating iron-overloaded patients with sickle cell disease or other transfusion-dependent anemias.
Researchers sought to determine whether TBI could help improve treatment with allogeneic blood or marrow transplantation in patients with severe hemoglobinopathies.
Researchers sought to determine what should be the primary treatment goal for patients with sickle cell disease.
Researchers sought to determine whether deferiprone could be a good option to treat pediatric patients with transfusion-dependent hemoglobinopathy.
Researchers sought to determine whether CRISPR-Cas9 gene therapy would be an effective treatment for patients with sickle cell disease or -thalassemia.
A single-center, open-label pilot study of 6 patients showed that the BCL11A gene is an effective target for HbF induction.
Investigators aimed to determine whether the sickle cell trait was associated with myocardial infarction or coronary heart disease among African American individuals.
Among patients with sickle cell disease, inhibition of BCL11A via gene knockdown is an effect target to induce fetal hemoglobin.
Preliminary results suggest that CRISPR-Cas9 editing of BCL11A may benefit patients with sickle cell disease and β-thalassemia by potentially eliminating vaso-occlusive episodes or the need for transfusion.
Investigators aimed to understand patient-reported effects of sickle cell disease on health, wellbeing, and disease management.
