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Molecules 2009, 14(3), 1304-1323; doi:10.3390/molecules14031304

Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos

*  and
Gene Tools, LLC, 1001 Summerton Way, Philomath, OR 97370 USA
* Author to whom correspondence should be addressed.
Received: 2 March 2009 / Revised: 23 March 2009 / Accepted: 24 March 2009 / Published: 25 March 2009
(This article belongs to the Special Issue Nucleic Acids)
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Antisense molecules do not readily cross cell membranes. This has limited the use of antisense to systems where techniques have been worked out to introduce the molecules into cells, such as embryos and cell cultures. Uncharged antisense bearing a group of guanidinium moieties on either a linear peptide or dendrimer scaffold can enter cells by endocytosis and subsequently escape from endosomes into the cytosol/nuclear compartment of cells. These technologies allow systemic administration of antisense, making gene knockdowns and splice modification feasible in adult animals; this review presents examples of such animal studies. Techniques developed with PPMOs, which are an arginine-rich cell-penetrating peptide linked to a Morpholino oligo, can also be performed using commercially available Vivo-Morpholinos, which are eight guanidinium groups on a dendrimeric scaffold linked to a Morpholino oligo. Antisense-based techniques such as blocking translation, modifying pre-mRNA splicing, inhibiting miRNA maturation and inhibiting viral replication can be conveniently applied in adult animals by injecting PPMOs or Vivo-Morpholinos.
Keywords: Antisense; Morpholino; PPMO; Vivo-Morpholino Antisense; Morpholino; PPMO; Vivo-Morpholino
This is an open access article distributed under the Creative Commons Attribution License (CC BY) which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Moulton, J.D.; Jiang, S. Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos. Molecules 2009, 14, 1304-1323.

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