TitleIn vivo delivery of morpholino oligos by cell-penetrating peptides.
Publication TypeJournal Article
Year of Publication2013
AuthorsMoulton, HM
JournalCurr Pharm Des
Date Published2013
KeywordsAnimals, Bioterrorism, Cell-Penetrating Peptides, Clinical Trials as Topic, Drug Delivery Systems, Gene Knockdown Techniques, Humans, Marburg Virus Disease, Morpholinos, Muscular Dystrophy, Duchenne, Tissue Distribution

Morpholino oligos (Morpholinos) are widely used tools for knocking down gene expression and are currently in a clinical trial for treatment of Duchene muscular dystrophy. A Morpholino analog has been in a clinical trial as a potential anti-bioterrorism agent for inhibiting replication of deadly Marburg viral infection. The cellular uptake of Morpholinos can been greatly increased by conjugation with cell-penetrating peptides (CPP). The use of the CPP-Morpholino conjugates (PPMOs) in vivo has been broadly demonstrated in viral, bacterial, genetic and other diseases. The following aspects of PPMOs will be discussed in this paper including chemistry, stability, antisense specificity, mechanism of cellular uptake, in vivo efficacy, tissue distribution, pharmacokinetics, toxicity and the human clinical trials. PPMOs are powerful research tools for studying gene function in animals and their properties are being improved as potential human therapeutic agents.

Alternate JournalCurr Pharm Des
PubMed ID23140456