通过结合基因补充和基因组编辑治疗脊髓性肌萎缩症的策略。
Mesh : Muscular Atrophy, Spinal / therapy genetics Animals Gene Editing / methods Survival of Motor Neuron 1 Protein / genetics CRISPR-Cas Systems Mice Genetic Therapy / methods Disease Models, Animal Humans Motor Neurons / metabolism pathology Mutation Male Female
来 源:
DOI:10.1038/s41467-024-50095-5
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