NACFC 2021 | W05: Approaches for Delivery of Nucleotide Based Therapeutics

Restoration of CFTR function with nucleotide-based therapeutics is an exciting next step towards a cystic fibrosis cure; however, delivery of the required therapeutic components into the right cells in vivo remains a major challenge. This workshop highlights progress made towards the delivery of nucleotide-based therapeutics in vitro and in vivo in both animal models and humans. The topics include novel vectors and formulations to deliver nucleotide-based therapeutics, antisense oligonucleotide, and premature termination codon suppression therapies, and cell and molecular biology relevant to CFTR regulation and function.

Originally recorded November 3, 2021.