Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos

@inproceedings{Moulton2009GeneKI,
  title={Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos},
  author={Jon D. Moulton and Shan Jiang},
  booktitle={Molecules},
  year={2009}
}
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… CONTINUE READING

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