Nusinersen 治疗未改变脊髓性肌萎缩症中 CSF 和脑实质内细胞介导的细胞毒性。
Mesh : Humans Oligonucleotides Muscular Atrophy, Spinal / drug therapy pathology genetics Motor Neurons / drug effects pathology metabolism Killer Cells, Natural / immunology drug effects Brain / pathology drug effects Female Male Survival of Motor Neuron 2 Protein / genetics CD8-Positive T-Lymphocytes / immunology drug effects Survival of Motor Neuron 1 Protein / genetics metabolism Single-Cell Analysis Cytotoxicity, Immunologic / drug effects Infant Child, Preschool Child Transcriptome
来 源:
DOI:10.1038/s41467-024-48195-3
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