Seamless Gene Correction in the Human Cystic Fibrosis Transmembrane Conductance Regulator Locus by Vector Replacement and Vector Insertion Events
Abstract
Background: Gene correction via homology directed repair (HDR) in patient-derived induced pluripotent stem (iPS) cells for regenerative medicine are becoming a more realistic approach to develop personalized and mutation-specific therapeutic strategies due to current developments in gene editing and iPSC technology. Cystic fibrosis (CF) is the most common inherited disease in the Caucasian population, caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Since CF causes significant multi-organ damage and with over 2,000 reported CFTR mutations, CF patients could be one prominent population benefiting from gene and cell therapies. When considering gene-editing techniques for clinical applications, seamless gene corrections of the responsible mutations, restoring native “wildtype” DNA sequence without remnants of drug selectable markers or unwanted DNA sequence changes, would be the most desirable approach.
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- Apr 06, 2022
- Source ID
- 10.3389/fgeed.2022.843885
Entities
People
- Cristina Barillà
- Dieter C. Gruenert
- Hirofumi Kai
- Keisuke Chosa
- Mary Ann Suico
- Michael Yao
- Orsetta Zuffardi
- R. Geoffrey Sargent
- Shingo Suzuki
- Tsuyoshi Shuto
- Yuet W. Kan
Organizations
- Japan Society for the Promotion of Science
- National Institutes of Health
- United States Department of Defense