Endocrine Abstracts

Introduction: Cushing’s disease is a devastating condition associated with a fivefold excess mortality. It is usually due to a small (few mm) benign corticotroph tumour in the pituitary expressing excess pro-opiomelanocortin (POMC), the peptide product of which, ACTH, drives excess secretion of cortisol from the adrenal. There is a clear clinical need for better treatment options.Background: We have designed, optimized and validated unique siRNAs to...

Background: Previously, we have demonstrated highly potent knockdown of POMC mRNA and significant reduction in secreted ACTH using siRNA, both in vitro and in vivo. Highly efficient delivery of siRNA to a target tissue is of critical importance for any therapeutic approach. Here we assess the suitability of a pH-sensitive polymer, PMPC-PDPA polymersomes encapsulating siRNA, as a novel delivery system as these have potential for tissue-directed delivery by ...