Journal
GENE THERAPY
Volume 14, Issue 8, Pages 682-689Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.gt.3302910
Keywords
non-viral gene delivery system; programmed packaging; octaarginine; multifunctional envelope-type nano device; in vivo topical application; hair growth
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Funding
- Intramural NIH HHS Funding Source: Medline
- NIEHS NIH HHS [Z01 ES071003-08] Funding Source: Medline
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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.
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