Molecular BiologybioRxivPreprint
Antisense oligonucleotide therapy for the common Stargardt disease type 1-causing variant in ABCA4
Melita Kaltak, Petra de Bruijn, Davide Piccolo…·Jim Swildens
This study demonstrates that antisense oligonucleotides can correct splicing defects in the ABCA4 c.5461-10T>C variant, restoring protein function in retinal organoids.
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