PNA-peptide conjugates as intracellular gene control agents.
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.
Ivanova
Gabriela D
GD
Medical Research Council, Laboratory of Molecular Biology, Hills Road, Cambridge CB2 0QH, UK.
Fabani
Martin M
MM
Arzumanov
Andrey A
AA
Abes
Rachida
R
Yin
Haifang
H
Lebleu
Bernard
B
Wood
Matthew
M
Gait
Michael J
MJ
D000818:Animals; D006367:HeLa Cells; D006801:Humans; D049409:Luciferases, Firefly / Q000096:biosynthesis; D051379:Mice; D035683:MicroRNAs / Q000037:antagonists & inhibitors* / Q000378:metabolism; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics; D020135:Peptide Nucleic Acids / Q000737:chemistry*; D012326:RNA Splicing; D055544:beta-Globins / Q000235:genetics
D035683:MicroRNAs; D020135:Peptide Nucleic Acids; D055544:beta-Globins; D049409:Luciferases, Firefly