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Journal ArticleDOI

Cell-penetrating peptides for the delivery of nucleic acids

Taavi Lehto, +2 more
- 20 Jun 2012 - 
- Vol. 9, Iss: 7, pp 823-836
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TLDR
This review will discuss recent advances of CPP-mediated delivery of nucleic acid-based cargo, concentrating on the delivery of plasmid DNA, splice-correcting ONs, and small-interfering RNAs.
Abstract
Introduction: Different gene therapy approaches have gained extensive interest lately and, after many initial hurdles, several promising approaches have reached to the clinics. Successful implementation of gene therapy is heavily relying on finding efficient measures to deliver genetic material to cells. Recently, non-viral delivery of nucleic acids and their analogs has gained significant interest. Among non-viral vectors, cell-penetrating peptides (CPPs) have been extensively used for the delivery of nucleic acids both in vitro and in vivo. Areas covered: In this review we will discuss recent advances of CPP-mediated delivery of nucleic acid-based cargo, concentrating on the delivery of plasmid DNA, splice-correcting ONs, and small-interfering RNAs. Expert opinion: CPPs have proved their potential as carriers for nucleic acids. However, similarly to other non-viral vectors, CPPs require further development, as efficient systemic delivery is still seldom achieved. To achieve this, CPPs should be modified...

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Citations
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Journal ArticleDOI

Brain Pathogenesis and Potential Therapeutic Strategies in Myotonic Dystrophy Type 1

TL;DR: In this paper, the authors summarize the current advances in the pathogenesis and pathology of central nervous system (CNS) deficits in myotonic dystrophy type 1 (DM1), and intervention measures currently being investigated are also highlighted, aiming to promote novel and cutting-edge therapeutic investigations.
Journal ArticleDOI

Effect of small molecule signaling in PepFect14 transfection.

TL;DR: The indicated small molecules interfere via still unidentified interactions in cell signaling, leading to an up-regulation of endocytosis and a higher efficacy in the delivery of short splice correcting oligonucleotides in complex with PepFect14.
Book ChapterDOI

Classes and Applications of Cell-Penetrating Peptides

TL;DR: It is complicated to classify the cell-penetrating peptides (CPPs) due to the definition obstacles, so here some possibilities are presented based on their type of origin, biochemical properties, and physico-chemical properties.
Journal ArticleDOI

Surface-Engineered Mucus Penetrating Nucleic Acid Delivery Systems with Cell Penetrating Peptides for the Lungs.

TL;DR: A mucus penetrating system for the delivery of both small RNA and plasmid DNA independently, which hypothesize that the hydrophilic coating of CS-A would reduce the surface charge and decrease the interaction with negatively charged mucins, while the conjugated mannitol residues would disrupt the mucin-mucin interaction or decrease the viscosity of mucus by increasing the influx of water into the mucus.
Journal ArticleDOI

Delivery of therapeutic small interfering RNA: The current patent-based landscape

TL;DR: In this article , an overall patent landscape of siRNA delivery was presented from the temporal, spatial, organizational, and technological dimensions, and a total of 11,509 patent documents from 3,309 patent families were collected, classified into 10 technological categories and comprehensively analyzed.
References
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Journal ArticleDOI

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Journal ArticleDOI

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