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Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos

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 pep...

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Autores principales: Moulton, Jon D., Jiang, Shan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Molecular Diversity Preservation International 2009
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6253989/
https://www.ncbi.nlm.nih.gov/pubmed/19325525
http://dx.doi.org/10.3390/molecules14031304
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author Moulton, Jon D.
Jiang, Shan
author_facet Moulton, Jon D.
Jiang, Shan
author_sort Moulton, Jon D.
collection PubMed
description 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.
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spelling pubmed-62539892018-11-30 Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos Moulton, Jon D. Jiang, Shan Molecules Review 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. Molecular Diversity Preservation International 2009-03-25 /pmc/articles/PMC6253989/ /pubmed/19325525 http://dx.doi.org/10.3390/molecules14031304 Text en © 2009 by the authors; licensee Molecular Diversity Preservation International, Basel, Switzerland. This article is an open-access article distributed under the terms and conditions of the Creative Commons Attribution license (http://creativecommons.org/licenses/by/3.0/).
spellingShingle Review
Moulton, Jon D.
Jiang, Shan
Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos
title Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos
title_full Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos
title_fullStr Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos
title_full_unstemmed Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos
title_short Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos
title_sort gene knockdowns in adult animals: ppmos and vivo-morpholinos
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6253989/
https://www.ncbi.nlm.nih.gov/pubmed/19325525
http://dx.doi.org/10.3390/molecules14031304
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