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Serum-stabilized PNA-internalization peptides (Pip) conjugated to PNA complementary to the 705 aberrant beta-globin splice site are able to correct splicing and increase luciferase production in Hela pLuc705 cells with sub microM EC(50) in the absence of a transfection agent. Inhibition of microRNA-122 in liver cells is achieved by treatment with complementary PNA containing just a few attached Lys residues, again without need of a transfection agent.

Original publication




Journal article


Nucleic Acids Symp Ser (Oxf)

Publication Date



31 - 32


Animals, HeLa Cells, Humans, Luciferases, Firefly, Mice, MicroRNAs, Muscular Dystrophy, Duchenne, Peptide Nucleic Acids, RNA Splicing, beta-Globins