Nanoparticles That Effectively Deliver Charge-Neutral Oligonucleotide Analog Drugs into Cells

Therapeutic oligonucleotide analogs represent a new and promising family of drugs that act on nucleic acid targets such as RNA or DNA; however, their effectiveness has been limited due to difficulty crossing the cell membrane. A new delivery approach based on cell-penetrating peptide nanoparticles can efficiently transport charge-neutral oligonucleotide analogs into cells, as reported online on January 16, 2015 in an open-access article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. In the article, entitled "Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides," Dr. Peter Järver form the Medical Research Council (MRC) on the Cambridge Biomedical Campus (UK) and coauthors also from the Cambridge Biomedical Campus (U.K.), and also from Karolinska University Hospital (Huddinge, Sweden), Stockholm University (Sweden), Alexandria University (Egypt), and University of Oxford (UK), note that while delivery systems exist to facilitate cell entry of negatively charged oligonucleotide drugs, these approaches are not effective for charge-neutral oligonucleotide analogs. The authors describe lipid-functionalized peptides that form a complex with charge-neutral morpholino oligonucleotides, enabling them to cross into cells and retain their biological activity. "The exploitation of phosphorodiamidate morpholinos represents an exciting approach to treating a number of therapeutic targets," says Nucleic Acid Therapeutics Executive Editor Graham C. Parker, Ph.D., The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit, Michigan, an who was not involved in the research.
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