Gene delivery of cystic fibrosis therapies with polymeric nanoparticles – UROP Symposium

Gene delivery of cystic fibrosis therapies with polymeric nanoparticles

Karen Reynoso

Research Mentor(s): Alexandra Piotrowski-Daspit
Program: CCSFP
Authors: Karen Reynoso, Alexandra Piotrowski-Daspit, PhD

Abstract

Nonsense mutations/premature termination codons (PTC) are those that convert a regular amino acid codon into a stop codon.This almost always results in a loss of function for the shortened protein. Cystic fibrosis (CF) is caused by mutations to the CF transmembrane conductance regulator (CFTR) gene encoding a chloride channel. Approximately 11% of CF mutations are caused by PTCs. Current small molecule therapies are not effective at treating CF caused by PTCs because they only target full length proteins. The use of anticodon edited (ACE) tRNAs to replace the stop codon in an effort to salvage the CFTR protein is a potential therapeutic. We used a polymer from the poly(amine-co-ester) (PACE) family to formulate nanoparticles for ACE-tRNA delivery to human bronchial epithelial cells harboring the W1282X CF PTC mutation.

lsa logoum logo