基因治疗医学

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. SMA is caused by mutations in the survival motor n...

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...