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

Medically reviewed by Gagandeep Brar, MDBeta thalassemia minor—also known as beta thalassemia trait or B thalassemia—is a ...

A family of Albanian descent had an unusual form of beta thalassemia. The father of two children with mild thalassemia major had normal red-cell morphology and normal levels of hemoglobins A 2 and ...

Thalassemia is a hereditary blood disorder causing anaemia due to reduced haemoglobin production. Early detection, awareness, ...

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 While gene therapy has emerged as a promising alternative for treating beta-thalassemia, hematopoietic stem cell transplantation (HSCT) remains the only widely ...

They altered lab-made embryos to remove the disease beta-thalassemia ... human genetic variants associated with disease are point mutations. "So base editing has the potential to directly correct ...

Stanley's battle began at just eight months old when he was diagnosed with beta-thalassemia major, the most severe form of the disease. His bone marrow could not produce sufficient red blood cells ...

Thalassemia is a rare inherited blood disorder caused by genetic mutations that lead to a reduced ... with transfusion-dependent alpha- or beta-thalassemia, achieving its primary endpoint.

The treatment for sickle cell disease and beta thalassemia is the first to be licensed using the gene-editing tool known as Crispr, for which its discoverers were awarded the Nobel prize in 2020.