List of 82 genes previously reported to be associated with neuromuscular disorder among the muscle genes with factor loadings whose absolute value is above 2.

NIAID Data Ecosystem40

Gene therapy in neuromuscular disorders

肌肉疾病基因治疗

病毒载体基因治疗

Abstract Monogenic neuromuscular disorders are potentially treatable through gene therapy. Using viral vectors, a therapeutic transgene aims to restore normal levels of a protein not produced by the d

Figshare2022-05-01 更新30

Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy

临床前药物筛选

肌肉疾病基因治疗

A readily available animal model is essential for rapidly identifying effective treatments for Duchenne muscular dystrophy (DMD), a devastating neuromuscular disorder caused by the lack of dystrophin

NIAID Data Ecosystem60

Effects of Pten knockout in dystrophic skeletal muscles of mdx mouse. Effects of Pten knockout in dystrophic skeletal muscles of mdx mouse

肌肉疾病基因治疗

骨骼肌转录组

Analysis of skeletal muscles with specific knockout (KO) of Phosphatase and tensin homolog (Pten) gene in an animal model of DMD (mdx mice). Pten knockout alleviates myofiber degeneration and restores

NIAID Data Ecosystem30