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 ...

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 ...

thalassemia refers specifically to the abnormal or absent manufacturing of alpha-globin chains. These are associated with more than 15 different genetic mutations. The severity of the clinical ...

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 Pharmaceuticals a significant new revenue source beyond cystic fibrosis, especially as ...

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.