Antisense Oligonucleotide Screening to Optimize the Rescue of the Splicing Defect Caused by the Recurrent Deep-Intronic ABCA4 Variant C.4539+2001g>a in Stargardt Disease
Genes（2019）
关键词
antisense oligonucleotides,Stargardt disease,inherited retinal diseases,splicing modulation,RNA therapy,ABCA4,iPSC-derived photoreceptor precursor cells
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