TL;DR: The data suggests that, by appropriate formulation of DNA complexes, complement activation can be minimized or even avoided, and should facilitate the search for DNA complex formulations appropriate for reproducible intravenous gene delivery.
Abstract: We have examined the complement-activating properties of synthetic cationic molecules and their complexes with DNA. Commonly used gene delivery vehicles include complexes of DNA with polyl...
TL;DR: The results suggest that expanded polyQ repeats in ATN1 may contribute to neurodegeneration via alterations in both protein aggregation and intracellular localization.