Correction of the Splicing Defect Caused by a Recurrent Variant in ABCA4 (C.769-784C>t) That Underlies Stargardt Disease
Cells（2022）
Key words
antisense oligonucleotides,ABCA4,Stargardt disease,inherited retinal disease,splicing modulation,RNA therapy,deep-intronic,HEK293T cells,patient-derived fibroblast cells,patient-derived photoreceptor precursor cells
AI Read Science
Must-Reading Tree
Example
Generate MRT to find the research sequence of this paper
Chat Paper
Summary is being generated by the instructions you defined