Figure - PMC

Skip to main content

An official website of the United States government

Here's how you know

Here's how you know

Official websites use .gov
A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS
A lock ( ) or https:// means you've safely connected to the .gov website. Share sensitive information only on official, secure websites.

View full-text article in PMC

. 2019 May 14;10(5):368. doi: 10.3390/genes10050368

Search in PMC
Search in PubMed
View in NLM Catalog
Add to search

© 2019 by the authors.

Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).

PMC Copyright notice

Antisense oligonucleotides (AONs)-mediated splicing alteration of CEP290 pre-mRNA to bypass protein truncation. An exonic mutation in CEP290 pre-mRNA (c.4723A > T, in red) introduces a PTC within exon 36 to CEP290 mRNA. Administration of AON (in black), targeting the splice donor site (H36D), is predicted to alter splicing by blocking the recognition of exon 36. Exclusion of exon 36 (CEP290^∆36) should allow bypassing protein truncation while maintaining the reading frame, and lead to the production of near full-length CEP290 protein.