Article
Adenine base editing reduces misfolded protein accumulation and toxicity in alpha-1 antitrypsin deficient patient iPSC-hepatocytes.
Molecular therapy : the journal of the American Society of Gene Therapy - 3 Nov 2021
Werder Rhiannon B, Kaserman Joseph E, Packer Michael S, Lindstrom-Vautrin Jonathan, Villacorta-Martin Carlos, Young Lauren E, Aratyn-Schaus Yvonne, Gregoire Francine, Wilson Andrew A
Abstract excerpt
Alpha-1 antitrypsin deficiency (AATD) is most commonly caused by the Z mutation, a single-base substitution that leads to AAT protein misfolding and associated liver and lung disease. In this study, we apply adenine base editors to correct the Z mutation in patient induced pluripotent stem cells (iPSCs) and iPSC-derived hepatocytes (iHeps). We demonstrate that correction of the Z mutation in patient iPSCs reduces...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.