Gene editing platforms for T-cell immunotherapy
Cell & Gene Therapy Insights 2019; 5(5), 705-718.
Genome editing holds the remarkable potential to transform medicine as a new therapeutic modality enabling correction of genetic defects and customization of genetic addition or ablation. The discovery and optimization of gene editing tools such as meganucleases, ZFNs, TALENs and CRISPR/Cas9 are advancing the prospects of clinical applications. Clinical trials using gene-edited T cells have been conducted or are in progress for the treatment of patients with HIV infection and various cancers. T cells engineered using combinations of gene editing and gene transfer technologies are also being investigated. The development of off-target detection methodologies and the establishment of efficient manufacturing platforms are essential to bring gene-edited T cells to the forefront of novel immunotherapies.