Octaarginine-modified multifunctional envelope-type nanoparticles for gene delivery.
This study describes a multifunctional envelope-type nano device (MEND) that mimics an envelope-type virus based on a novel packaging strategy. MEND particles contain a DNA core packaged into a lipid envelope modified with an octaarginine peptide. The peptide mediates internalization via macropinocytosis, which avoids lysosomal degradation. MEND-mediated transfection of a luciferase expression plasmid achieved comparable efficiency to adenovirus-mediated transfection, with lower associated cytotoxicity. Furthermore, topical application of MEND particles containing constitutively active bone morphogenetic protein (BMP) type IA receptor (caBmpr1a) gene had a significant impact on hair growth in vivo. These data demonstrate that MEND is a promising non-viral gene delivery system that may provide superior results to existing non-viral gene delivery technologies.
Khalil
I A
IA
Graduate School of Pharmaceutical Sciences, Hokkaido University, Sapporo, Hokkaido, Japan.
Kogure
K
K
Futaki
S
S
Hama
S
S
Akita
H
H
Ueno
M
M
Kishida
H
H
Kudoh
M
M
Mishina
Y
Y
Kataoka
K
K
Yamada
M
M
Harashima
H
H
D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D002460:Cell Line; D015870:Gene Expression; D005818:Genetic Engineering; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008156:Luciferases / Q000032:analysis / Q000235:genetics; D051379:Mice; D008817:Mice, Mutant Strains; D018613:Microscopy, Confocal; D053758:Nanoparticles; D009842:Oligopeptides / Q000235:genetics*; D012867:Skin / Q000378:metabolism / Q000821:virology; D014162:Transfection / Q000379:methods*; D001616:beta-Galactosidase / Q000032:analysis / Q000235:genetics
D009842:Oligopeptides; C448619:octaarginine; D008156:Luciferases; D001616:beta-Galactosidase