GeneticsbioRxivPreprint
Splice-switching antisense oligonucleotides correct cryptic exon inclusion and restore SDCCAG8 protein in Bardet-Biedl Syndrome
Kelleen E McEntee, Bailey L McCurdy, Austin A Larson…·Sujatha Jagannathan
This paper identifies ASOs that correct cryptic exon inclusion in SDCCAG8, restoring protein expression in BBS patients.
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