Gene Therapy to the Rescue

Beta thalassemia is a genetic blood disorder that affects the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. Individuals with beta thalassemia have a deficiency in beta globin, a subunit of hemoglobin... Read More ››

Cargo size is key Duchenne Muscular Dystrophy (DMD) is a genetic disease that affects ~1 in every 5,000 people. The disease results in muscle weakness and loss, eventually resulting in death due to a genetic mutation in the dystrophin gene. Dystrophi... Read More ››