Multifunctional envelope-type nano device (MEND) as a non-viral gene delivery system

Kogure, Kentaro; Akita, Hidetaka; Yamada, Yuma; Harashima, Hideyoshi

doi:10.1016/j.addr.2007.10.007

Cited by 181 publications

(103 citation statements)

References 53 publications

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“…Recently, a nanodevice possessing the desired characteristics of an ideal nonviral macromolecule delivery method has been developed based on the concept of ''programmed packaging'' (72). Multifunctional envelope-type nanodevice (MEND) represents a novel strategy of cellular delivery of macromolecules using STR-R8 as an essential component.…”

Section: Delivery Of Proteins As Cpp-cargo Complexmentioning

confidence: 99%

“…MITO-porter is one such molecule for mitochondrial delivery for drug therapy based on the concept of STR-R8 MEND. It is a liposome-based nanocarrier that can introduce cargos into mitochondria via membrane fusion (72).…”

Section: Delivery Of Cpp-cargo Complex To Mitochondriamentioning

confidence: 99%

“…This can be a major drawback for their use in therapeutics and other pharmacological application. Activation of CPPs only in the presence of specific cells or development of novel nanodevices such as MEND that can target CPPs to a specific cell type are some of the potential solutions (72,97). Also, it should be taken into consideration that optimal binding of nucleic acid will be different from proteins or drugs owing to the inherent chemical differences of these molecules.…”

Section: Cellular Internalization Mechanism Of Cpp-driven Cargo Complexmentioning

confidence: 99%

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Cell‐penetrating peptides: Nanocarrier for macromolecule delivery in living cells

2010

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SummaryNovel classes and applications of cell-penetrating peptides (CPPs) are being constantly discovered since they were first identified 2 decades ago. These short cationic peptides (nanomolecules) either by covalent binding or by noncovalent binding can traverse cell membranes and deliver a variety of molecules that are unable to overcome the permeability barrier in their own capacity. The ability of the CPPs to deliver variety of macromolecules, such as oligonucleotides, therapeutic drugs, proteins, and medical imaging agents, by forming nanoparticulate carriers in a range of cells has led them to emerge as a potential tool for both macromolecule delivery application and to gain insight into the fundamentals of mechanism of cellular uptake across the plasma membrane. This review explores the recent advances, challenges, and future prospects in the field of CPP-mediated cargo delivery in mammalian and plant cells. Studies have been conducted into the peptide chemistry and stability of CPP-macromolecular complexes. Most of the CPPs have been shown to be nontoxic and do not interfere with the functionality of the macromolecules delivered across the cell membrane. The mechanism of uptake of CPP-cargo complexes and the uptake of CPPs alone across the plasma membrane remains unresolved. As the world of CPPs is rapidly advancing in both mammalian and plant system, there is a promising future for the various applications of transduction and transfection into intact cells.

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Section: Delivery Of Proteins As Cpp-cargo Complexmentioning

confidence: 99%

Section: Delivery Of Cpp-cargo Complex To Mitochondriamentioning

confidence: 99%

Section: Cellular Internalization Mechanism Of Cpp-driven Cargo Complexmentioning

confidence: 99%

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Cell‐penetrating peptides: Nanocarrier for macromolecule delivery in living cells

2010

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“…Even through in vitro experiments have been reported, actual studies of mitochondrial gene delivery are very few [8][9][10][11]. The use of a variety of applications for nuclear gene delivery in vitro/in vivo have been reported [11][12][13][14][15][16], in attempts to accelerate progress in the field of nuclear gene therapy. Thus, successful mitochondrial gene transfer would largely contribute to mitochondrial gene therapy.…”

Section: Introductionmentioning

confidence: 99%

Localization of exogenous DNA to mitochondria in skeletal muscle following hydrodynamic limb vein injection

Yasuzaki

Yamada

Kanefuji

et al. 2013

Journal of Controlled Release

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ABSTRACT.Mitochondrial genetic disorders are a major cause of mitochondrial diseases. It is therefore likely that mitochondrial gene therapy will be useful for the treatment of such diseases. Here, we report on the possibility of mitochondrial gene delivery in skeletal muscle using hydrodynamic limb vein (HLV) injection. The HLV injection procedure, a useful method for transgene expression in skeletal muscle, involves the rapid injection of a large volume of naked plasmid DNA (pDNA) into the distal vein of a limb. We hypothesized that the technique could be used to deliver pDNA not only to nuclei but also mitochondria, since cytosolic pDNA that is internalized by the method may be able to overcome mitochondrial membrane. We determined if pDNA could be delivered to myofibrillar mitochondria by HLV injection by PCR analysis. Mitochondrial toxicity assays showed that the HLV injection had no influence on mitochondrial function. These findings indicate that HLV injection promises to be a useful technique for in vivo mitochondrial gene delivery.

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“…Transgene expression has long been proposed as an innovative therapeutic strategy for directly treating diseases at the level of genes, and considerable efforts have been made to improve the efficiency of protein production [1][2][3][4][5][6][7]. To successfully achieve this, an efficient and targeted gene delivery system is necessary.…”

Section: Introductionmentioning

confidence: 99%

Post-nuclear gene delivery events for transgene expression by biocleavable polyrotaxanes

Yamada¹,

Nomura²,

Harashima³

et al. 2012

Biomaterials

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2 ABSTRACTA quantitative comparison between nuclear DNA release from carriers and their transfection activity would be highly useful for improving the effectiveness of non-viral gene vectors. We previously reported that, for condensed DNA particles, a close relationship exists between the efficiency of DNA release and transfection activity, when biocleavable polyrotaxanes (DMAE-SS-PRX), in which the cationic density can be easily controlled. In this study, we first investigated the efficiencies of DNA release from condensed DNA particles with various types of DMAE-SS-PRX. The findings indicate that an optimal cationic density in DMAE-SS-PRX exists for DNA release. We then packaged condensed DNA particles in a multifunctional envelope-type nano device (MEND), and evaluated their transfection activities. The results showed that the transfection activity was increased and this increase was, to some extent, dependent on the efficiency of the DNA release. However, transfection activity decreased, when the value for the efficiency of DNA release was higher than a certain value. An investigation of the fate of intranuclear DNA indicated that a very high efficiency of DNA release has a positive influence on transcription, however, it would inhibit the post-transcription process; nuclear mRNA export, translation and related processes.Such information provides a new viewpoint for the development of cationic polymer-based vectors.

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Multifunctional envelope-type nano device (MEND) as a non-viral gene delivery system

Cited by 181 publications

References 53 publications

Cell‐penetrating peptides: Nanocarrier for macromolecule delivery in living cells

Cell‐penetrating peptides: Nanocarrier for macromolecule delivery in living cells

Localization of exogenous DNA to mitochondria in skeletal muscle following hydrodynamic limb vein injection

Post-nuclear gene delivery events for transgene expression by biocleavable polyrotaxanes

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