articleScienceFeb 6, 2020GREEN OA

CRISPR-engineered T cells in patients with refractory cancer

EAEdward A. StadtmauerJAJoseph A. FraiettaMMMegan M. DavisADAdam D. CohenKWKristy Weber

University of Pennsylvania · Parker Institute for Cancer Immunotherapy · +2 more institutions

PubMed
Indexed incrossrefpubmed

Abstract

CRISPR takes first steps in humans CRISPR-Cas9 is a revolutionary gene-editing technology that offers the potential to treat diseases such as cancer, but the effects of CRISPR in patients are currently unknown. Stadtmauer et al. report a phase 1 clinical trial to assess the safety and feasibility of CRISPR-Cas9 gene editing in three patients with advanced cancer (see the Perspective by Hamilton and Doudna). They removed immune cells called T lymphocytes from patients and used CRISPR-Cas9 to disrupt three genes ( TRAC, TRBC , and PDCD1 ) with the goal of improving antitumor immunity. A cancer-targeting transgene, NY-ESO-1, was also introduced to recognize tumors. The engineered cells were administered to…

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Authors

41

Topics & keywords

Topics
Keywords
  • CRISPR
  • Genome editing
  • T-cell receptor
  • Cas9
  • Computational biology
  • Multiplex
  • Gene
  • Cancer
UN Sustainable Development Goals
  • Good health and well-being
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