关键词: Becker muscular dystrophy Duchenne muscular dystrophy antisense therapy dystrophin restoration early exon skipping treatment exon 6-8 skipping morpholinos multiple exon skipping neonatal dystrophic dogs systemic treatment
Mesh : Animals Animals, Newborn Disease Models, Animal Dogs Dystrophin / genetics metabolism Exons / genetics Morpholinos / genetics Muscle, Skeletal / metabolism pathology Muscular Dystrophy, Duchenne / genetics therapy Oligonucleotides, Antisense / genetics Reading Frames / genetics
来 源:
DOI:10.1016/j.ymthe.2018.10.011
PDF(Sci-hub)
PDF(Pubmed)