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Title: Knocking out barriers to engineered cell activity

Abstract

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.

Authors:
 [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
Publication Date:
Research Org.:
Lawrence Berkeley National Lab. (LBNL), Berkeley, CA (United States)
Sponsoring Org.:
USDOE Office of Science (SC)
OSTI Identifier:
1633261
Grant/Contract Number:  
AC02-05CH11231
Resource Type:
Accepted Manuscript
Journal Name:
Science
Additional Journal Information:
Journal Volume: 367; Journal Issue: 6481; Journal ID: ISSN 0036-8075
Publisher:
AAAS
Country of Publication:
United States
Language:
English
Subject:
60 APPLIED LIFE SCIENCES

Citation Formats

Hamilton, Jennifer R., and Doudna, Jennifer A. Knocking out barriers to engineered cell activity. United States: N. p., 2020. Web. https://doi.org/10.1126/science.aba9844.
Hamilton, Jennifer R., & Doudna, Jennifer A. Knocking out barriers to engineered cell activity. United States. https://doi.org/10.1126/science.aba9844
Hamilton, Jennifer R., and Doudna, Jennifer A. Thu . "Knocking out barriers to engineered cell activity". United States. https://doi.org/10.1126/science.aba9844. https://www.osti.gov/servlets/purl/1633261.
@article{osti_1633261,
title = {Knocking out barriers to engineered cell activity},
author = {Hamilton, Jennifer R. and Doudna, Jennifer A.},
abstractNote = {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.},
doi = {10.1126/science.aba9844},
journal = {Science},
number = 6481,
volume = 367,
place = {United States},
year = {2020},
month = {2}
}

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Works referenced in this record:

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