The clinical potential of gene editing as a tool to engineer cell-based therapeutics

Clin Transl Med. 2020 Feb 7;9(1):15. doi: 10.1186/s40169-020-0268-z.


The clinical application of ex vivo gene edited cell therapies first began a decade ago with zinc finger nuclease editing of autologous CD4+ T-cells. Editing aimed to disrupt expression of the human immunodeficiency virus co-receptor gene CCR5, with the goal of yielding cells resistant to viral entry, prior to re-infusion into the patient. Since then the field has substantially evolved with the arrival of the new editing technologies transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR), and the potential benefits of gene editing in the arenas of immuno-oncology and blood disorders were quickly recognised. As the breadth of cell therapies available clinically continues to rise there is growing interest in allogeneic and off-the-shelf approaches and multiplex editing strategies are increasingly employed. We review here the latest clinical trials utilising these editing technologies and consider the applications on the horizon.

Keywords: CRISPR–Cas9; Cell therapy; Clinical trial; HIV; Oncology; TALEN; Zinc finger nuclease.

Publication types

  • Review