دورية أكاديمية
أعرض في EDS

Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering.

التفاصيل البيبلوغرافية
العنوان: Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering.
المؤلفون: Hamilton JR; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA., Tsuchida CA; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA; University of California, Berkeley-University of California, San Francisco Graduate Program in Bioengineering, University of California, Berkeley, Berkeley, CA 94720, USA., Nguyen DN; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA; Division of Infectious Diseases, Department of Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Department of Microbiology and Immunology, University of California, San Francisco, San Francisco, CA 94143, USA; Diabetes Center, University of California, San Francisco, San Francisco, CA 94143, USA; Gladstone Institutes, University of California, San Francisco, San Francisco, CA 94158, USA., Shy BR; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA; Department of Microbiology and Immunology, University of California, San Francisco, San Francisco, CA 94143, USA; Diabetes Center, University of California, San Francisco, San Francisco, CA 94143, USA; Gladstone Institutes, University of California, San Francisco, San Francisco, CA 94158, USA; Department of Laboratory Medicine, University of California, San Francisco, San Francisco, CA 94143, USA., McGarrigle ER; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA; Howard Hughes Medical Institute, University of California, Berkeley, Berkeley, CA 94720, USA., Sandoval Espinoza CR; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA., Carr D; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA; Division of Infectious Diseases, Department of Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Department of Microbiology and Immunology, University of California, San Francisco, San Francisco, CA 94143, USA; Diabetes Center, University of California, San Francisco, San Francisco, CA 94143, USA; Gladstone Institutes, University of California, San Francisco, San Francisco, CA 94158, USA., Blaeschke F; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA; Division of Infectious Diseases, Department of Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Department of Microbiology and Immunology, University of California, San Francisco, San Francisco, CA 94143, USA; Diabetes Center, University of California, San Francisco, San Francisco, CA 94143, USA; Gladstone Institutes, University of California, San Francisco, San Francisco, CA 94158, USA., Marson A; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA; Division of Infectious Diseases, Department of Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Department of Microbiology and Immunology, University of California, San Francisco, San Francisco, CA 94143, USA; Diabetes Center, University of California, San Francisco, San Francisco, CA 94143, USA; Gladstone Institutes, University of California, San Francisco, San Francisco, CA 94158, USA; Parker Institute for Cancer Immunotherapy, San Francisco, CA 94158, USA; Chan Zuckerberg Biohub, San Francisco, CA 94158, USA; Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco, San Francisco, CA 94158, USA., Doudna JA; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94720, USA; University of California, Berkeley-University of California, San Francisco Graduate Program in Bioengineering, University of California, Berkeley, Berkeley, CA 94720, USA; Gladstone Institutes, University of California, San Francisco, San Francisco, CA 94158, USA; Howard Hughes Medical Institute, University of California, Berkeley, Berkeley, CA 94720, USA; California Institute for Quantitative Biosciences (QB3), University of California, Berkeley, Berkeley, CA 94720, USA; Molecular Biophysics and Integrated Bioimaging Division, Lawrence Berkeley National Laboratory, Berkeley, CA 94720, USA; Department of Chemistry, University of California, Berkeley, Berkeley, CA 94720, USA. Electronic address: doudna@berkeley.edu.
المصدر: Cell reports [Cell Rep] 2021 Jun 01; Vol. 35 (9), pp. 109207.
نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
اللغة: English
بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101573691 Publication Model: Print Cited Medium: Internet ISSN: 2211-1247 (Electronic) NLM ISO Abbreviation: Cell Rep Subsets: MEDLINE
أسماء مطبوعة: Original Publication: [Cambridge, MA] : Cell Press, c 2012-
مواضيع طبية MeSH: Cell Engineering* , Gene Transfer Techniques* , Transgenes*, CRISPR-Cas Systems/*genetics, A549 Cells ; CD4-Positive T-Lymphocytes/metabolism ; CRISPR-Associated Protein 9/metabolism ; Gene Editing ; HIV-1/physiology ; Humans ; Jurkat Cells ; Lentivirus/genetics ; Receptors, Chimeric Antigen/metabolism ; Ribonucleoproteins/metabolism ; Virion/metabolism ; env Gene Products, Human Immunodeficiency Virus
مستخلص: As genome engineering advances cell-based therapies, a versatile approach to introducing both CRISPR-Cas9 ribonucleoproteins (RNPs) and therapeutic transgenes into specific cells would be transformative. Autologous T cells expressing a chimeric antigen receptor (CAR) manufactured by viral transduction are approved to treat multiple blood cancers, but additional genetic modifications to alter cell programs will likely be required to treat solid tumors and for allogeneic cellular therapies. We have developed a one-step strategy using engineered lentiviral particles to introduce Cas9 RNPs and a CAR transgene into primary human T cells without electroporation. Furthermore, programming particle tropism allows us to target a specific cell type within a mixed cell population. As a proof-of-concept, we show that HIV-1 envelope targeted particles to edit CD4 + cells while sparing co-cultured CD8 + cells. This adaptable approach to immune cell engineering ex vivo provides a strategy applicable to the genetic modification of targeted somatic cells in vivo.
Competing Interests: Declaration of interests The Regents of the University of California have patents issued and pending for CRISPR technologies on which the authors are co-inventors. J.A.D. is a cofounder of Caribou Biosciences, Editas Medicine, Scribe Therapeutics, Intellia Therapeutics, and Mammoth Biosciences. J.A.D. is a scientific advisory board member of Caribou Biosciences, Intellia Therapeutics, eFFECTOR Therapeutics, Scribe Therapeutics, Mammoth Biosciences, Synthego, Algen Biotechnologies, Felix Biosciences, and Inari. J.A.D. is a Director at Johnson & Johnson and Tempus and has research projects sponsored by Biogen, Pfizer, AppleTree Partners, and Roche. A.M. is a compensated co-founder, member of the boards of directors, and a member of the scientific advisory boards of Spotlight Therapeutics and Arsenal Biosciences. A.M. was a compensated member of the scientific advisory board at PACT Pharma and was a compensated advisor to Juno Therapeutics and Trizell. A.M. owns stock in Arsenal Biosciences, Spotlight Therapeutics, and PACT Pharma. A.M. has received honoraria from Merck and Vertex, a consulting fee from AlphaSights, and is an investor in and informal advisor to Offline Ventures. The Marson lab has received research support from Juno Therapeutics, Epinomics, Sanofi, GlaxoSmithKline, Gilead, and Anthem. J.R.H. has consulted for Scribe Therapeutics. All of the other authors have no competing interests.
(Copyright © 2021 The Author(s). Published by Elsevier Inc. All rights reserved.)
التعليقات: Comment in: Immunol Cell Biol. 2021 Aug;99(7):677-679. (PMID: 34231252)
References: ACS Chem Biol. 2018 Feb 16;13(2):376-382. (PMID: 29019396)
Elife. 2014 Apr 24;3:e01911. (PMID: 24843011)
PLoS One. 2018 Apr 4;13(4):e0195558. (PMID: 29617431)
Gene Ther. 2011 Sep;18(9):936-41. (PMID: 21451580)
Nat Rev Cancer. 2016 Aug 23;16(9):566-81. (PMID: 27550819)
Hum Gene Ther. 2010 May;21(5):589-602. (PMID: 20039782)
Nat Biotechnol. 2017 May;35(5):431-434. (PMID: 28191903)
Nat Biotechnol. 2016 Mar;34(3):339-44. (PMID: 26789497)
Nat Commun. 2019 Jan 3;10(1):45. (PMID: 30604748)
Sci Rep. 2017 Apr 7;7(1):737. (PMID: 28389661)
Br Med Bull. 2001;58:43-59. (PMID: 11714623)
Science. 2020 Feb 28;367(6481):. (PMID: 32029687)
Nat Biotechnol. 2019 Dec;37(12):1425-1434. (PMID: 31160723)
Nat Nanotechnol. 2019 Oct;14(10):974-980. (PMID: 31501532)
Gene Ther. 2016 Jul;23(7):627-33. (PMID: 27052803)
Nucleic Acids Res. 2020 Aug 20;48(14):8178-8187. (PMID: 32619241)
Nucleic Acids Res. 2014 Feb;42(4):e28. (PMID: 24270790)
Curr Gene Ther. 2005 Aug;5(4):387-98. (PMID: 16101513)
Proc Natl Acad Sci U S A. 2018 May 8;115(19):4903-4908. (PMID: 29686087)
Nat Biotechnol. 2020 Jan;38(1):44-49. (PMID: 31819258)
Nature. 2020 Feb;578(7794):229-236. (PMID: 32051598)
Mol Ther. 2010 Oct;18(10):1814-21. (PMID: 20628360)
Genome Res. 2014 Jun;24(6):1020-7. (PMID: 24696462)
N Engl J Med. 2019 Mar 7;380(10):947-959. (PMID: 30855744)
Nat Chem Biol. 2018 Feb;14(2):112-117. (PMID: 29200207)
Cell. 2020 Apr 2;181(1):46-62. (PMID: 32243795)
Cell. 2017 Feb 9;168(4):724-740. (PMID: 28187291)
Annu Rev Med. 2017 Jan 14;68:139-152. (PMID: 27860544)
J Virol. 1995 Jun;69(6):3389-98. (PMID: 7745685)
Nucleic Acids Res. 2011 Sep 1;39(17):7610-9. (PMID: 21715375)
JCI Insight. 2020 Feb 27;5(4):. (PMID: 31999649)
Nature. 2017 May 24;545(7655):423-431. (PMID: 28541315)
Nat Commun. 2020 Jun 26;11(1):3232. (PMID: 32591530)
Front Immunol. 2021 Mar 23;12:639818. (PMID: 33833759)
J Am Chem Soc. 2018 May 30;140(21):6596-6603. (PMID: 29668265)
Nature. 2018 Jul;559(7714):405-409. (PMID: 29995861)
Science. 2012 Aug 17;337(6096):816-21. (PMID: 22745249)
Nucleic Acids Res. 2019 Sep 26;47(17):e99. (PMID: 31299082)
Cancer Immunol Immunother. 2019 Mar;68(3):365-377. (PMID: 30523370)
Sci Rep. 2012;2:359. (PMID: 22496955)
Nat Biotechnol. 2020 Jul;38(7):845-855. (PMID: 32601435)
Mol Ther. 2020 Nov 4;28(11):2320-2339. (PMID: 32979309)
معلومات مُعتمدة: K08 AI153767 United States AI NIAID NIH HHS; RM1 HG009490 United States HG NHGRI NIH HHS; L40 AI140341 United States AI NIAID NIH HHS; U01 AI142817 United States AI NIAID NIH HHS; United States HHMI Howard Hughes Medical Institute
فهرسة مساهمة: Keywords: CAR-T cells; CRISPR delivery; CRISPR-Cas9; precision genome editing; viral engineering; virus-like particles
المشرفين على المادة: 0 (Receptors, Chimeric Antigen)
0 (Ribonucleoproteins)
0 (env Gene Products, Human Immunodeficiency Virus)
EC 3.1.- (CRISPR-Associated Protein 9)
تواريخ الأحداث: Date Created: 20210602 Date Completed: 20220211 Latest Revision: 20220509
رمز التحديث: 20221213
مُعرف محوري في PubMed: PMC8236216
DOI: 10.1016/j.celrep.2021.109207
PMID: 34077734
قاعدة البيانات: MEDLINE
الوصف
تدمد:2211-1247
DOI:10.1016/j.celrep.2021.109207