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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

Cell-penetrating peptides: 20 years later, where do we stand?

TL;DR: This review is dedicated to CPP fundamentals, with an emphasis on the molecular requirements and mechanism of their entry into eukaryotic cells.
Journal ArticleDOI

Cell-Penetrating Peptides : Design, Synthesis, and Applications

TL;DR: The intrinsic property of cell-penetrating peptides to deliver therapeutic molecules to cells and tissues in a nontoxic manner has indicated that they may be potential components of future drugs and disease diagnostic agents.
Journal ArticleDOI

The delivery of therapeutic oligonucleotides.

TL;DR: A variety of current approaches for enhancing the delivery of oligonucleotides including molecular scale targeted ligand-oligonucleotide conjugates, lipid- and polymer-based nanoparticles, antibody conjugate and small molecules that improve oligon nucleotide delivery are examined.
Journal ArticleDOI

Non viral vectors in gene therapy- an overview.

TL;DR: This review will see in detail about various types of non-viral vectors highlighting promising development and recent advances that had improved the non-Viral gene transfer efficiency of translating from "Bench to bedside".
Journal ArticleDOI

Cellular Uptake of Nanoparticles versus Small Molecules: A Matter of Size.

TL;DR: Particular attention is paid to approaches that allow conditional regulation of the cell internalization process using external triggers, such as activable cell penetrating peptides, due to the impact these systems may have in drug delivery and sensing applications.
References
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Journal ArticleDOI

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

TL;DR: Three RNA-based therapeutic technologies exploiting various oligonucleotides that bind to RNA by base pairing in a sequence-specific manner yet have different mechanisms of action and effects are discussed.
Journal ArticleDOI

Cell penetration by transportan

TL;DR: The cell-penetrating ability of transportan is not restricted by cell type, but seems to be a general feature of this peptide.
Journal ArticleDOI

State-of-the-art gene-based therapies: the road ahead.

TL;DR: Improvements in the gene transfer vectors used in therapeutic trials have led to substantial clinical successes in patients with serious genetic conditions, such as immunodeficiency syndromes, blindness and some cancer types.
Journal ArticleDOI

Erratum: Knocking down barriers: advances in siRNA delivery

TL;DR: The authors would like to clarify that, following a Phase I/II trial in patients with wet age-related macular degeneration, the siRNA drug candidate PF-4523655 (RTP801i-14) is now being studied in a Phase II trial for diabetic macular oedema conducted by Pfizer in collaboration with Quark.
Journal ArticleDOI

A new peptide vector for efficient delivery of oligonucleotides into mammalian cells

TL;DR: This new strategy of oligonucleotide delivery into cultured cells based on a peptide vector offers several advantages compared to other commonly used approaches of delivery including efficiency, stability and absence of cytotoxicity, and is proposed as a powerful tool for potential development in gene and antisense therapy.
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