CRISPR-engineered T cells in patients with refractory cancer
Edward A. StadtmauerAbramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAJoseph A. FraiettaAbramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAMegan M. DavisCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAAdam D. CohenAbramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAKristy WeberAbramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAEric LancasterDepartment of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAPatricia ManganDivision of Hematology-Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAIrina KulikovskayaCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAMinnal GuptaCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAFang ChenCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USALifeng TianCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAVanessa GonzalezCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAJun XuCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAIn-Young JungCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAJ. Joseph MelenhorstCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAGabriela PlesaCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAJoanne SheaCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USATina MatlawskiCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAAmanda CerviniCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAAvery L. GaymonCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAStephanie DesjardinsCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAAnne LamontagneCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAJanuary Salas-McKeeCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAAndrew D. FesnakCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USADonald L. SiegelCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USABruce L. LevineCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAJulie K. JadlowskyCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USARegina M. YoungCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAAnne ChewCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAWei‐Ting HwangDepartment of Biostatistics, Epidemiology and Informatics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAElizabeth O. HexnerAbramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USABeatriz M. CarrenoCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAChristopher L. NoblesDepartment of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAFrederic D. BushmanDepartment of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USAKevin R. ParkerCenter for Personal Dynamic Regulomes, Stanford University School of Medicine, Stanford, CA, USAYanyan QiDepartment of Pathology, Stanford University School of Medicine, Stanford, CA, USAAnsuman T. SatpathyCenter for Personal Dynamic Regulomes, Stanford University School of Medicine, Stanford, CA, USAHoward Y. ChangCenter for Personal Dynamic Regulomes, Stanford University School of Medicine, Stanford, CA, USAYangbing ZhaoCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USASimon F. LaceyCenter for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USACarl H. JuneAbramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
2020en
ABI
Аннотация
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
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