Article
In Vitro Validation of a CRISPR-Mediated CFTR Correction Strategy for Preclinical Translation in Pigs
17 May 2019
Abstract excerpt
Early efforts in cystic fibrosis (CF) gene therapy faced major challenges in delivery efficiency and sustained therapeutic gene expression. Recent advancements in engineered site-specific endonucleases such as clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 make permanent CF transmembrane conductance regulator ( CFTR ) gene correction possible. However, because of safety concerns of the...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
