用反义寡核苷酸 sepofarsen 单次治疗后可持久改善视力 : 一例病例报告。
Mesh : Antigens, Neoplasm / genetics metabolism Cell Cycle Proteins / genetics metabolism Ciliopathies / genetics therapy Cytoskeletal Proteins / genetics metabolism Genetic Therapy / methods Humans Leber Congenital Amaurosis / genetics physiopathology therapy Oligonucleotides, Antisense / therapeutic use Photoreceptor Cells / metabolism Vision, Ocular / physiology Visual Fields / physiology
来 源:
DOI:10.1038/s41591-021-01297-7
PDF(Pubmed)