Norah Awad has lived with beta thalassemia major, a severe blood disorder that has required monthly transfusions since she ...

While gene therapy has emerged as a promising alternative for treating beta-thalassemia ... pointing out that better HLA typing is a major reason. However, a "big quantum leap" was the marked ...

LentiGlobin BB305 aims to treat beta-thalassemia major and severe sickle cell disease ... a single treatment of the gene therapy corrected the main genetic defect which causes the condition.

The therapy – which is based on CD34+ stem cells taken from patients that are modified to carry a working gene for beta-globin, a haemoglobin component that is deficient in thalassemia patients ...

Thalassemia and sickle cell anaemia are common genetic disorders in India. Early testing, prenatal screening, and improved ...

the first CRISPR/Cas9 gene-editing therapy in the UAE. This achievement opens new horizons and offers innovative treatment for patients suffering from sickle cell disease (SCD) and transfusion ...

Gene therapy and gene editing are on the cutting ... in December 2023 and for treating transfusion-dependent beta thalassemia (TDT) just six weeks later. Vertex presented additional positive ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

India has around 50 million (5 crore) carriers of the ß (beta) thalassemia trait ... U.S. has shown promising results for gene therapy for thalassemia major. • FDA-Approved Therapies: Trials ...

Thalassemia and Sickle Cell Anaemia are common genetic disorders, with options for treatment and prevention available.