Nature1y
Genetic Mutation
Molecules of sickle-cell hemoglobin stick to one another ... often resulting in a frameshift One form of beta-thalassemia Deletion One or more nucleotides is "skipped" during replication or ...
Nature2y
Thalassemia: the long road from the bedside through the laboratory to the community
Because of the paucity of red blood cell precursors capable of synthesizing hemoglobin in the blood of normal people or people with thalassemia ... diversity in the structure of the α-globin ...
MedPage Today6d
Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
2 Minute Medicine7d
Beti-cel gene therapy achieves high rates of transfusion independence in severe β-thalassemia
There were no serious adverse events or treatment-related mortalities. Evidence Rating Level: 2 (Good) Study Rundown: ...