In vivo gene silencing following non-invasive siRNA delivery into the skin using a novel topical formulation.

ABSTRACT: Therapeutics based on short interfering RNAs (siRNAs), which act by inhibiting the expression of target transcripts, represent a novel class of potent and highly specific next-generation treatments for human skin diseases. Unfortunately, the intrinsic barrier properties of the skin combined with the large size and negative charge of siRNAs make epidermal delivery of these macromolecules quite challenging. To help evaluate the in vivo activity of these therapeutics and refine delivery strategies we generated an innovative reporter mouse model that predominantly expresses firefly luciferase (luc2p) in the paw epidermis--the region of murine epidermis that most closely models the tissue architecture of human skin. Combining this animal model with state-of-the-art live animal imaging technique

SUBMITTER: Hegde V 

PROVIDER: S-EPMC4275573 | biostudies-literature | 2014 Dec

REPOSITORIES: biostudies-literature