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Knocking out barriers to engineered cell activity

Journal Article · · Science
 [1];  [2]
  1. Univ. of California, Berkeley, CA (United States). Howard Hughes Medical Institute
  2. Univ. of California, Berkeley, CA (United States). Howard Hughes Medical Institute, California Institute for Quantitative Biosciences (QB3), Innovative Genomics Institute; Lawrence Berkeley National Lab. (LBNL), Berkeley, CA (United States); Univ. of California, San Francisco, CA (United States). Gladstone Institutes
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Engineered T cell therapies are revolutionizing cancer treatment by achieving long-lasting remission in blood-related cancers, such as leukemia and lymphoma. These therapies involve removal of patient T cells, “reprogramming” them to attack cancer cells, and then transferring them back into the patient. Targeted gene inactivation (knockout) using CRISPR-Cas9 can enhance T cell activity (1, 2) and has the potential to expand cell therapy applications. Until now, it has been unknown whether CRISPR-Cas9–edited T cells would be tolerated and thrive once reinfused into a human. On page 1001 of this issue, Stadtmauer et al. (3) present data from a phase 1 clinical trial (designed to test safety and feasibility) on the first cancer patients treated with CRISPR-Cas9–modified T cells. The findings represent an important advance in the therapeutic application of gene editing and highlight the potential to accelerate development of cell-based therapies.

Research Organization:
Lawrence Berkeley National Laboratory (LBNL), Berkeley, CA (United States)
Sponsoring Organization:
USDOE Office of Science (SC)
Grant/Contract Number:
AC02-05CH11231
OSTI ID:
1633261
Journal Information:
Science, Journal Name: Science Journal Issue: 6481 Vol. 367; ISSN 1095-9203; ISSN 0036-8075
Publisher:
AAASCopyright Statement
Country of Publication:
United States
Language:
English

References (10)

CRISPR-Cas9-mediated multiplex gene editing in CAR-T cells journal December 2016
Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection journal February 2017
Reprogramming human T cell function and specificity with non-viral genome targeting journal July 2018
Identification of preexisting adaptive immunity to Cas9 proteins in humans journal January 2019
CRISPR/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells journal April 2017
Optimized RNP transfection for highly efficient CRISPR/Cas9-mediated gene knockout in primary T cells journal February 2018
CAR T cell immunotherapy for human cancer journal March 2018
CRISPR-engineered T cells in patients with refractory cancer journal February 2020
Multiplex Genome Editing to Generate Universal CAR T Cells Resistant to PD1 Inhibition journal November 2016
A Pilot Trial of the Combination of Transgenic NY-ESO-1–reactive Adoptive Cellular Therapy with Dendritic Cell Vaccination with or without Ipilimumab journal December 2018

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60 APPLIED LIFE SCIENCES