期刊
CLINICAL AND TRANSLATIONAL MEDICINE
卷 9, 期 1, 页码 -出版社
SPRINGEROPEN
DOI: 10.1186/s40169-020-0268-z
关键词
Cell therapy; Clinical trial; CRISPR-Cas9; HIV; Oncology; TALEN; Zinc finger nuclease
资金
- Guy's & St. Thomas' Charity
- Cancer Research UK [MDPA C48390/A21153]
- Wellcome/EPSRC Centre for Medical Engineering at KCL [WT 203148/Z/16/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.
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