维甲酸相关的孤儿受体 α 是一种遗传修饰剂 , 可在 Stargardt 病和干性 AMD 的小鼠模型中挽救视网膜变性。
Mesh : Animals Humans Mice ATP-Binding Cassette Transporters / genetics metabolism Dependovirus / genetics Disease Models, Animal Genetic Therapy / methods Geographic Atrophy / genetics metabolism therapy Macular Degeneration / genetics metabolism therapy Mice, Knockout Nuclear Receptor Subfamily 1, Group F, Member 1 / genetics metabolism Retinal Degeneration / genetics therapy metabolism Stargardt Disease / genetics
来 源:
DOI:10.1038/s41434-024-00455-z
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