Beta-thalassemia is a potentially serious genetic disorder that a person can pass on to their children. However, with the right treatment, an individual can often live a typical and healthy life.

With beta thalassemia, your body doesn’t make enough hemoglobin, an important protein used by red blood cells. ... A s tem cell transplant and gene therapy may also be options. Symptoms.

Thalassemia is a genetic blood disorder that impacts the ability of the blood to get oxygen to the body’s organs. Learn about the signs, how it’s diagnosed & treated.

Casgevy is now the second gene therapy approved for beta thalassemia, following the 2022 FDA nod for Zynteglo from Bluebird Bio.Zynteglo carries a $2.8 million wholesale price, but the biotech ...

One gene mutation: Beta-thalassemia minor (trait) — usually asymptomatic or mild anaemia. Two gene mutations: Beta-thalassemia major (Cooley’s anaemia) — severe anaemia requiring regular ...

In a stroke of luck, or divine providence as Shelby’s parents see it, this gene therapy for her disease, beta thalassemia, had been approved just the year before.

Beta thalassemia can be due to mutations that convert A to G in a particular location in the HBB gene, the scientists noted in the study, in which they used base editing to try to convert G back to A.

Wanda Sihanath, 22, received gene therapy for beta thalassemia. Now a college senior at Arizona State University, she says she has been transfusion-free for the past four years.

Wanda Sihanath, 22, received gene therapy for beta thalassemia. Now a college senior at Arizona State University, she says she has been transfusion-free for the past four years.

Beta thalassemia can be due to mutations that convert A to G in a particular location in the HBB gene, the scientists noted in the study, in which they used base editing to try to convert G back to A.