There are three main types of beta thalassemia: minor, intermedia, and major. Mutations (variants) in the HBB gene (the gene that controls the production of the beta-globin proteins that make up ...

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 ...

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 ...

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 ...

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.

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 ...

It can generate billions in revenue from both acute and possibly chronic pain. If approved, it would give Vertex ...

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 ...

Orna Therapeutics and Vertex will use lipid nanoparticle system to develop gene-editing therapies for people with SCD and TDT ...