There are three main types of beta thalassemia: minor, intermedia, and major. Mutations (variants) in the HBB gene (the gene ...

How do mutations happen, and how do they influence ... often resulting in a frameshift One form of beta-thalassemia Deletion One or more nucleotides is "skipped" during replication or otherwise ...

Opens in a new tab or window An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires life-long blood transfusions for survival ...

Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Several manufacturers are developing novel treatments for the disease, including Vertex, which has partnered ...

On January 21st, the Thalassemia International Federation (TIF) and BGI Genomics jointly hosted an educational webinar focused on thalassemia prevention in high-prevalence regions. Experts emphasized ...

is a severe form of beta-thalassemia, a hereditary blood disorder caused by mutations in the HBB gene, which encodes the beta-globin component of hemoglobin. TDT and SCD have high unmet medical needs.

thalassemia refers specifically to the abnormal or absent manufacturing of alpha-globin chains. These are associated with more than 15 different genetic mutations. The severity of the clinical ...

YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204, an investigational therapy for the ...

Extra iron can damage the heart, liver, and endocrine system. Someone with beta thalassemia has a change (or mutation) in the beta globin gene that causes less beta globin to be made than typical. The ...