CRISPR-engineered T cells in patients with refractory cancer-Reference-Cited by-同舟云学术

CRISPR-engineered T cells in patients with refractory cancer

Published:2020-02-28 Issue:6481 Volume:367 Page:
ISSN:0036-8075
Container-title:Science
language:en
Short-container-title:Science

Author:

Stadtmauer Edward A.¹²^ORCID,Fraietta Joseph A.²³⁴⁵⁶^ORCID,Davis Megan M.⁵⁶^ORCID,Cohen Adam D.¹²^ORCID,Weber Kristy L.²⁷,Lancaster Eric⁸,Mangan Patricia A.¹,Kulikovskaya Irina⁵^ORCID,Gupta Minnal⁵^ORCID,Chen Fang⁵,Tian Lifeng⁵^ORCID,Gonzalez Vanessa E.⁵,Xu Jun⁵,Jung In-young⁴⁵,Melenhorst J. Joseph³⁵⁶,Plesa Gabriela⁵^ORCID,Shea Joanne⁵^ORCID,Matlawski Tina⁵^ORCID,Cervini Amanda⁵^ORCID,Gaymon Avery L.⁵^ORCID,Desjardins Stephanie⁵^ORCID,Lamontagne Anne⁵^ORCID,Salas-Mckee January⁵,Fesnak Andrew⁵⁶^ORCID,Siegel Donald L.⁵⁶^ORCID,Levine Bruce L.⁵⁶^ORCID,Jadlowsky Julie K.⁵,Young Regina M.⁵^ORCID,Chew Anne⁵,Hwang Wei-Ting⁹^ORCID,Hexner Elizabeth O.¹²^ORCID,Carreno Beatriz M.³⁵⁶,Nobles Christopher L.⁴^ORCID,Bushman Frederic D.⁴^ORCID,Parker Kevin R.¹⁰^ORCID,Qi Yanyan¹¹,Satpathy Ansuman T.¹⁰¹¹^ORCID,Chang Howard Y.¹⁰¹²^ORCID,Zhao Yangbing⁵⁶^ORCID,Lacey Simon F.⁵⁶^ORCID,June Carl H.²³⁵⁶^ORCID

Affiliation:

1. Division of Hematology-Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

2. Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

3. Parker Institute for Cancer Immunotherapy, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

4. Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

5. Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

6. Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

7. Department of Orthopaedic Surgery, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

8. Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

9. Department of Biostatistics, Epidemiology and Informatics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

10. Center for Personal Dynamic Regulomes, Stanford University School of Medicine, Stanford, CA, USA.

11. Department of Pathology, Stanford University School of Medicine, Stanford, CA, USA.

12. Howard Hughes Medical Institute, Stanford University School of Medicine, Stanford, CA, USA.

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 patients and were well tolerated, with durable engraftment observed for the study duration. These encouraging observations pave the way for future trials to study CRISPR-engineered cancer immunotherapies. Science , this issue p. eaba7365 ; see also p. 976

Funder

Office of Extramural Research, National Institutes of Health

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

Reference55 articles.

1. Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease.

2. A New Class of Medicines through DNA Editing

3. Gene Editing ofCCR5in Autologous CD4 T Cells of Persons Infected with HIV

4. CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia

5. A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity

Cited by 996 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Recent advances and applications of the CRISPR-Cas system in the gene therapy of blood disorders;Gene;2024-12

2. Precision Genome Editing Techniques in Gene Therapy: Current State and Future Prospects;Current Gene Therapy;2024-10

3. Therapeutic peptides targeting intracellular molecules;European Polymer Journal;2024-10

4. CRISPRoff epigenetic editing for programmable gene silencing in human cells without DNA breaks;2024-09-09

5. A Digital Microfluidic Platform for the Microscale Production of Functional Immune Cell Therapies;2024-09-08