Exon skipping via chimeric antisense U1 snRNAs to correct Retinitis Pigmentosa GTPase-Regulator (RPGR) splice defect.
Giuseppina Covello, Gehan H. Ibrahim, Niccolò Bacchi, Simona Casarosa, 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.
Nucleic Acid Therapeutics · Feb 15, 2022Peer-reviewedMolecular Biology