Endosomolytic nano-polyplex platform technology for cytosolic peptide delivery to inhibit pathological vasoconstriction

BC Evans, KM Hocking, KV Kilchrist, ES Wise… - ACS …, 2015 - ACS Publications
BC Evans, KM Hocking, KV Kilchrist, ES Wise, CM Brophy, CL Duvall
ACS nano, 2015ACS Publications
A platform technology has been developed and tested for delivery of intracellular-acting
peptides through electrostatically complexed nanoparticles, or nano-polyplexes, formulated
from an anionic endosomolytic polymer and cationic therapeutic peptides. This delivery
platform has been initially tested and optimized for delivery of two unique vasoactive
peptides, a phosphomimetic of heat shock protein 20 and an inhibitor of MAPKAP kinase II,
to prevent pathological vasoconstriction (ie, vasospasm) in human vascular tissue. These …
A platform technology has been developed and tested for delivery of intracellular-acting peptides through electrostatically complexed nanoparticles, or nano-polyplexes, formulated from an anionic endosomolytic polymer and cationic therapeutic peptides. This delivery platform has been initially tested and optimized for delivery of two unique vasoactive peptides, a phosphomimetic of heat shock protein 20 and an inhibitor of MAPKAP kinase II, to prevent pathological vasoconstriction (i.e., vasospasm) in human vascular tissue. These peptides inhibit vasoconstriction and promote vasorelaxation by modulating actin dynamics in vascular smooth muscle cells. Formulating these peptides into nano-polyplexes significantly enhances peptide uptake and retention, facilitates cytosolic delivery through a pH-dependent endosomal escape mechanism, and enhances peptide bioactivity in vitro as measured by inhibition of F-actin stress fiber formation. In comparison to treatment with the free peptides, which were endowed with cell-penetrating sequences, the nano-polyplexes significantly increased vasorelaxation, inhibited vasoconstriction, and decreased F-actin formation in the human saphenous vein ex vivo. These results suggest that these formulations have significant potential for treatment of conditions such as cerebral vasospasm following subarachnoid hemorrhage. Furthermore, because many therapeutic peptides include cationic cell-penetrating segments, this simple and modular platform technology may have broad applicability as a cost-effective approach for enhancing the efficacy of cytosolically active peptides.
ACS Publications