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

Autor: Candice Ashmore-Harris, Gilbert O. Fruhwirth
Jazyk: angličtina
Rok vydání: 2020
Předmět:
Zdroj: Clinical and Translational Medicine, Vol 9, Iss 1, Pp 1-22 (2020)
Druh dokumentu: article
ISSN: 2001-1326
DOI: 10.1186/s40169-020-0268-z
Popis: Abstract 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.
Databáze: Directory of Open Access Journals
Nepřihlášeným uživatelům se plný text nezobrazuje