Molecular BiologyNucleic Acid TherapeuticsPeer-reviewed
Exon skipping via chimeric antisense U1 snRNAs to correct Retinitis Pigmentosa GTPase-Regulator (RPGR) splice defect.
Giuseppina Covello, Gehan H. Ibrahim, Niccolò Bacchi…·Michela A. Denti
The paper demonstrates the use of chimeric antisense U1 snRNAs to skip an aberrant exon in RPGR, correcting a splicing defect in retinitis pigmentosa.
16 citations