The SMN protein localizes to both the cytoplasm and the nuclear bodies. One hallmark of spinal muscular atrophy is the failure of SMN to localize within these nuclear bodies.
结果显示 EVs 能有效将 SMN 蛋白递送至细胞,该成果为 SMA 治疗带来新希望 ... 《Scientific Reports》期刊上发表了题为 “Extracellular vesicles efficiently deliver survival motor neuron protein to cells in culture” 的论文。这篇论文在脊髓性肌萎缩症(Spinal ...
This protein is critical for motor neurons to make proteins and grow the "wires" that send signals out to the muscles. When the SMN1 gene is mutated, the body cannot make enough SMN protein ...
The scientists found that the child had higher levels of the SMN protein in their bloodstream, compared to babies normally born with the condition. They seemed to have lower levels of nerve damage ...
The three diagnostic tests for identifying genetic mutations associated with SMA met new European Union regulatory ...
The doctors found that the infant had higher levels of SMN protein and less nerve damage than other babies born with SMA type 1. In the months since her birth has shown no signs of abnormal muscle ...