A Precision Therapy Approach for Retinitis Pigmentosa 11 Using Splice-Switching Antisense Oligonucleotides to Restore the Open Reading Frame of PRPF31

A Precision Therapy Approach for Retinitis Pigmentosa 11 Using Splice-Switching Antisense Oligonucleotides to Restore the Open Reading Frame of PRPF31

Retinitis pigmentosa 11 is an untreatable, dominantly inherited retinal disease caused by heterozygous mutations in pre-mRNA processing factor 31 <i>PRPF31</i>. The expression level of <i>PRPF31</i> is linked to incomplete penetrance in affected families; mutation carriers wi...

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Bibliographic Details
Main Authors: Janya Grainok, Ianthe L. Pitout, Fred K. Chen, Samuel McLenachan, Rachael C. Heath Jeffery, Chalermchai Mitrpant, Sue Fletcher
Format: Article
Language:English
Published: MDPI AG 2024-03-01
Series:International Journal of Molecular Sciences
Subjects:
retinitis pigmentosa
splicing factor
antisense oligonucleotides
exon skipping
precision medicine
Online Access:https://www.mdpi.com/1422-0067/25/6/3391

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https://www.mdpi.com/1422-0067/25/6/3391

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