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Targeted antisense oligonucleotide therapy rescues PRPF31 expression in retinitis pigmentosa caused by a splicing mutation

βœ‡Molecular Therapy
By: Poulami Banik Β· Felix Zimmann Β· Prasoon K. Thakur Β· Lubica Dudakova Β· KateΕ™ina VečerkovΓ‘ Β· Ondrej Kostov Β· Marvin H. Caruthers Β· Michal KolΓ‘Ε™ Β· Inka KrejčířovΓ‘ Β· Marie Vajter Β· Petra Liskova Β· TomΓ‘Ε‘ BΓ‘rta Β· David StanΔ›k
Stanek and colleagues uncover a novel PRPF31 intronic mutation that disrupts splicing and lowers protein levels. Targeted antisense oligonucleotides restore normal splicing and boost PRPF31 expression in patient-derived RPE, highlighting a potential therapeutic strategy for retinitis pigmentosa.
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