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Int. J. Mol. Sci. 2016, 17(2), 263; doi:10.3390/ijms17020263

Intracellular Delivery of Proteins with Cell-Penetrating Peptides for Therapeutic Uses in Human Disease

1
Department of Pathology, University of Washington, Seattle, WA 98109, USA
2
Department of Medicine, Division of Cardiology, University of Washington, Seattle, WA 98109, USA
*
Author to whom correspondence should be addressed.
Academic Editor: Jagdish Singh
Received: 7 January 2016 / Revised: 4 February 2016 / Accepted: 16 February 2016 / Published: 22 February 2016
(This article belongs to the Special Issue Cell-Penetrating Peptides)
View Full-Text   |   Download PDF [216 KB, uploaded 22 February 2016]

Abstract

Protein therapy exhibits several advantages over small molecule drugs and is increasingly being developed for the treatment of disorders ranging from single enzyme deficiencies to cancer. Cell-penetrating peptides (CPPs), a group of small peptides capable of promoting transport of molecular cargo across the plasma membrane, have become important tools in promoting the cellular uptake of exogenously delivered proteins. Although the molecular mechanisms of uptake are not firmly established, CPPs have been empirically shown to promote uptake of various molecules, including large proteins over 100 kiloDaltons (kDa). Recombinant proteins that include a CPP tag to promote intracellular delivery show promise as therapeutic agents with encouraging success rates in both animal and human trials. This review highlights recent advances in protein-CPP therapy and discusses optimization strategies and potential detrimental effects. View Full-Text
Keywords: enzyme replacement therapy (ERT); cell-penetrating peptides (CPPs); enzyme deficiency; protein therapeutics enzyme replacement therapy (ERT); cell-penetrating peptides (CPPs); enzyme deficiency; protein therapeutics
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Dinca, A.; Chien, W.-M.; Chin, M.T. Intracellular Delivery of Proteins with Cell-Penetrating Peptides for Therapeutic Uses in Human Disease. Int. J. Mol. Sci. 2016, 17, 263.

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