Journal Update 3: CRISPR-engineered T cells in patients with refractory cancer

CRISPR provides a powerful tool to modify genes to treat a variety of illnesses. This paper presents the first human trials to use CRISPR edited t-cells to fight cancer. Specifically, the team deleted two genes (TRAC, TRBC) to reduce misparing of t-cell receptors and to enhance the synthetic, cancer specific transgene (NY-ESO-1). The team also removed a third gene (PDCD1) to improve anti-tumor immunity. While the study was not aiming to treat cancer, it did show the viability for using this method to treat future patients. Using three patients with refractory cancer, they showed that modified t-cells persisted for up to nine months, providing the framework for potential future therapy. 

Stadtmauer EA, Fraietta JA, Davis MM, Cohen AD, Weber KL, Lancaster E, Mangan PA, Kulikovskaya I, Gupta M, Chen F, Tian L. CRISPR-engineered T cells in patients with refractory cancer. Science. 2020 Feb 28;367(6481).

Ryan McRae
Science