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Octaarginine-modified multifunctional envelope-type nanoparticles for gene delivery.


ABSTRACT: 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.

SUBMITTER: Khalil IA 

PROVIDER: S-EPMC1847622 | biostudies-literature | 2007 Apr

REPOSITORIES: biostudies-literature

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Octaarginine-modified multifunctional envelope-type nanoparticles for gene delivery.

Khalil I A IA   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  

Gene therapy 20070201 8


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 cytot  ...[more]

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