Gene Therapy to the Rescue

CAR-T cell therapy is rapidly becoming a clinically viable option for cancer treatment and innovations in the field are on the rise. Read More ››

Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder characterized by the loss of motor neurons in the spinal cord and brainstem, leading to progressive muscle weakness and atrophy. Read More ››

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. Read More ››

화물 크기가 핵심 Duchenne 근이영양증(DMD)은 5,000명당 ~1명에게 영향을 미치는 유전질환입니다. Read More ››