关键词: AAV ALS ASO CRISPR RNAi amyotrophic lateral sclerosis clinical trial gene delivery gene therapy
Mesh : Amyotrophic Lateral Sclerosis / genetics therapy C9orf72 Protein / genetics Dependovirus / genetics Genetic Therapy / methods Humans Oligonucleotides, Antisense / genetics therapeutic use
来 源:
DOI:10.1016/j.ymthe.2021.04.008
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