Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2014 Feb;24(1):37-47.
doi: 10.1089/nat.2013.0454. Epub 2013 Oct 30.

A chemical view of oligonucleotides for exon skipping and related drug applications

Peter Järver  1 , Liz O'Donovan, Michael J Gait
Affiliations
Review

A chemical view of oligonucleotides for exon skipping and related drug applications

Peter Järver et al. Nucleic Acid Ther. 2014 Feb.
No abstract available

PubMed Disclaimer

Figures

<b>FIG. 1.</b>
FIG. 1.
Structures of repeating units of analogues of oligonucleotides (ONs) used in exon skipping and related applications. (R=O or S) (A) Charge-negative ONs. (B) Charge-neutral ONs.
See this image and copyright information in PMC

References

    1. AARTSMA-RUS A., KAMAN W.E., BREMMER-BOUT M., JANSON A.A.M., DEN DUNNEN J.T., VAN OMMEN G.B., and VAN DEUTEKOM J.C.T. (2004). Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells. Gene Ther. 11, 1391–1398 - PubMed
    1. ABES S., TURNER J.J., IVANOVA G.D., OWEN D., WILLIAMS D., ARZUMANOV A., CLAIR P., GAIT M.J., and LEBLEU B. (2007). Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide. Nucleic Acid Res. 35, 4495–4502 - PMC - PubMed
    1. ALAM M.R., DIXIT V., KANG H., LI Z.-B., CHEN X., TREJO J., FISHER M., and JULIANO R.L. (2008). Intracellular delivery of an anionic antisense oligonucleotide via receptor mediated endocystosis. Nucleic Acids Res. 36, 2764–2776 - PMC - PubMed
    1. ALTER J., LOU F., RABINOWITZ A., YIN H., ROSENFELD J., WILTON S.D., PARTRIDGE T.A., and LU Q.L. (2006). Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nature Med. 12, 175–177 - PubMed
    1. AOKI Y., YOKOTA T., NAGATA T., NAKAMURA A., TANIHATA J., SAITO T., DUGUEZ S.M.R., NAGARAJU K., HOFFMAN E.P., PARTRIDGE T., et al. (2012). Bodywide skipping of exons 45–55 in dystrophic mdx52 mice by systemic antisense delivery. Proc. Nat. Acad. Sci. U. S. A. 109, 13763–13768 - PMC - PubMed

Publication types

MeSH terms