Article
Efficient and allele-specific genome editing of disease loci in human iPSCs.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2015
Smith Cory, Abalde-Atristain Leire, He Chaoxia, Brodsky Brett R, Braunstein Evan M, Chaudhari Pooja, Jang Yoon-Young, Cheng Linzhao, Ye Zhaohui
Abstract excerpt
Efficient and precise genome editing is crucial for realizing the full research and therapeutic potential of human induced pluripotent stem cells (iPSCs). Engineered nucleases including CRISPR/Cas9 and transcription activator like effector nucleases (TALENs) provide powerful tools for enhancing gene-targeting efficiency. In this study, we investigated the relative efficiencies of CRISPR/Cas9 and TALENs in human...
Topics
- Alleles
- Bacterial Proteins
- Base Sequence
- CRISPR-Associated Protein 9
- Cell Line
- Clustered Regularly Interspaced Short Palindromic Repeats
- DNA End-Joining Repair
- Endonucleases
- Gene Expression
- Genome, Human