在小型猪中静脉和鞘内递送 AAV9 介导的 ARSA 的安全性和有效性。
关键词: adeno-associated virus arylsulfatase A central nervous system gene therapy metachromatic leukodystrophy neurodegeneration peripheral nervous system
Mesh : Humans Animals Swine Cerebroside-Sulfatase / genetics metabolism Swine, Miniature Neurodegenerative Diseases Leukodystrophy, Metachromatic / genetics metabolism Central Nervous System / metabolism Esterases
来 源:
DOI:10.3390/ijms24119204
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