Peptide-guided gene delivery

Martin, Molly E.; Rice, Kevin G.

doi:10.1208/aapsj0901003

Cited by 302 publications

(251 citation statements)

References 99 publications

(98 reference statements)

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“…However, marginal amounts of proteins were still detected inside the cells even with a high concentration of inhibitors, suggesting the possibility of another transduction mechanism that might be used such as binding to sialic acid, macropinocytosis, or direct penetration. Because the NLSs of many proteins are composed of positive charge-rich sequences, it is likely that more human-derived CPPs will be identified through the analysis of NLSs as CPP candidates (Martin and Rice, 2007). These CPPs might also have a similar cell-penetrating mechanism and efficient intracellular delivery as LPIN-CPP.…”

Section: Discussionmentioning

confidence: 99%

Identification of a Novel Cell-Penetrating Peptide from Human Phosphatidate Phosphatase LpIN3

Lim

Kim

et al. 2012

Molecules and Cells

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Section: Discussionmentioning

confidence: 99%

Identification of a Novel Cell-Penetrating Peptide from Human Phosphatidate Phosphatase LpIN3

Lim

Kim

et al. 2012

Molecules and Cells

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“…13 Different systems have been proposed and tested for nonviral gene delivery based on DNA condensation and protection. 12,14,15 However, the drawback of this therapeutic approach is often the lack of a successful in vivo application. There is clearly a need to develop highly efficient, chemical, nonviral gene carriers that can protect DNA and persist under physiological in vivo conditions.…”

Section: Introductionmentioning

confidence: 99%

Fatty acid–spermine conjugates as DNA carriers for nonviral in vivo gene delivery

et al. 2009

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The lack of efficient in vivo gene delivery is a well-known shortcoming of nonviral delivery vectors, in particular of chemical vectors. We developed a series of novel nonviral carriers for plasmid-based in vivo gene delivery. This new transport device is based on the assembly of DNA plasmids with synthetic derivatives of naturally occurring moleculesfatty acid-spermine conjugates (or lipospermines). We tested the ability of these fatty acid conjugates to interact with plasmid DNA (pDNA) and found that they formed DNA nanocomplexes, which are protected from DNase I degradation. This protection was shown to directly correlate with the length of the aliphatic component. However, this increase in the length of the hydrocarbon chain resulted in increased toxicity. The cationic lipids used for transfection typically have a C 16 and C 18 hydrocarbon chain. Interestingly, toxicity studies, together with further characterization studies, suggested that the two most suitable candidates for in vivo delivery are those with the shortest hydrocarbon chain, butanoyl-and decanoylspermine. Morphological characterization of DNA nanocomplexes resulting from these lipospermines showed the formation of a homogenous population, with the diameter ranging approximately from 40 to 200 nm. Butanoylspermine was found to be the most promising carrier from this series, resulting in a significantly increased gene expression, in relation to naked plasmid, in both tissues herein targeted (dermis and M. tibialis anterior). Thus, we established a correlation between the in vitro properties of the ensuing DNA nanocarriers and their efficient in vivo gene expression.

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“…Indeed, cytotoxicity was observed when the length of the PLL increased. [46] On the other hand, a low molecular weight PLL (MW < 3kDa) could not form stable complexes. [47] However, high molecular weight PLL was more suitable for gene delivery through systemic injection.…”

Section: Lipid-based Non-viral Vectorsmentioning

confidence: 99%

Different strategies of gene delivery for treatment of cancer and other disorders

Agi

Mosaferi

Khatamsaz

et al. 2016

JST

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Gene therapy is the gene transfer into host cells for treatment of acquired and genetic disorders. For this purpose, there are a wide variety of gene delivery methods with special properties including viral and non-viral vectors. The non-viral methods use physical forces or chemical compounds (natural or synthetic) to transfer DNA into a cell. The efficiency of the non-viral gene therapy depends on conquering four different intra-and extra-cellular barriers such as cellular uptake, endosomal escape, nuclear entry, and gene expression. Among various gene carriers, some viral vectors such as Adenovirus, Lentivirus, Vaccinia as well as gene gun and lipofection achieved to clinical trials. In this mini-review, we briefly describe different approaches for gene delivery and their applications in various phases of clinical trials.

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Peptide-guided gene delivery

Cited by 302 publications

References 99 publications

Identification of a Novel Cell-Penetrating Peptide from Human Phosphatidate Phosphatase LpIN3

Identification of a Novel Cell-Penetrating Peptide from Human Phosphatidate Phosphatase LpIN3

Fatty acid–spermine conjugates as DNA carriers for nonviral in vivo gene delivery

Different strategies of gene delivery for treatment of cancer and other disorders

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