PepFects and NickFects for the Intracellular Delivery of Nucleic Acids

Nucleic acids can be utilized in gene therapy to restore, alter, or silence gene functions. In order to reveal the biological activity nucleic acids have to reach their intracellular targets by passing through the plasma membrane, which is impermeable for these large and negatively charged molecules. Cell-penetrating peptides (CPPs) condense nucleic acids into nanoparticles using non-covalent complexation strategy and mediate their delivery into the cell, whereas the physicochemical parameters of the nanoparticles determine the interactions with the membranes, uptake mechanism, and subsequent intracellular fate. The nanoparticles are mostly internalized by endocytosis that leads to the entrapment of them in endosomal vesicles. Therefore design of new CPPs that are applicable for non-covalent complex formation strategy and harness endosomolytic properties is highly vital. Here we demonstrate that PepFects and NickFects are efficient vectors for the intracellular delivery of various nucleic acids.This chapter describes how to form CPP/pDNA nanoparticles, evaluate stable nanoparticles formation, and assess gene delivery efficacy.

Pärnaste, Ly (författare)
Hällbrink, MattiasStockholms universitet,Institutionen för neurokemi(Swepub:su)mhall (författare)
Langel, ÜloStockholms universitet,Institutionen för neurokemi,Tartu University, Estonia(Swepub:su)ulo (författare)
Stockholms universitetInstitutionen för neurokemi (creator_code:org_t)

Ingår i:Cell-Penetrating PeptidesNew York : Springer, s. 303-31597814939280579781493928064

Av författaren/redakt...: Arukuusk, Piret; Pärnaste, Ly; Hällbrink, Matti ...; Langel, Ülo

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