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Mol Ther. 2021 Nov 02; doi: 10.1016/j.ymthe.2021.10.026. Epub 2021 Nov 02.

Disruption of HIV-1 co-receptors CCR5 and CXCR4in primary human T cells and hematopoietic stemand progenitor cells using base editing.

Molecular therapy : the journal of the American Society of Gene Therapy

Friederike Knipping, Gregory A Newby, Cindy R Eide, Amber N McElroy, Sarah C Nielsen, Kyle Smith, Yongxing Fang, Tatjana I Cornu, Caroline Costa, Alejandra Gutierrez-Guerrero, Samuel P Bingea, Colby J Feser, Benjamin Steinbeck, Keli L Hippen, Bruce R Blazar, Anton McCaffrey, Claudio Mussolino, Els Verhoeyen, Jakub Tolar, David R Liu, Mark J Osborn

Affiliations

  1. Medical School, Department of Pediatrics, Division of Blood and Marrow Transplant & Cellular Therapy, University of Minnesota, Minneapolis, MN 55455, USA.
  2. Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, MA 02138, USA.
  3. Institute for Transfusion Medicine and Gene Therapy, Medical Center, University of Freiburg, Freiburg 79106, Germany; Center for Chronic Immunodeficiency, Medical Center, University of Freiburg, Freiburg 79106, Germany; MD Program, Faculty of Medicine, University of Freiburg, Freiburg 79106, Germany.
  4. Institute for Transfusion Medicine and Gene Therapy, Medical Center, University of Freiburg, Freiburg 79106, Germany; Center for Chronic Immunodeficiency, Medical Center, University of Freiburg, Freiburg 79106, Germany; Faculty of Medicine, University of Freiburg 79106, Germany.
  5. CIRI - International Center for Infectiology Research, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, Ecole Normale Supérieure de Lyon, Université Lyon, F-69007 Lyon, France.
  6. TriLink BioTechnologies, San Diego, CA 92121, USA.
  7. CIRI - International Center for Infectiology Research, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, Ecole Normale Supérieure de Lyon, Université Lyon, F-69007 Lyon, France; Université Côte d'Azur, INSERM, C3M, 06204 Nice, France.
  8. Medical School, Department of Pediatrics, Division of Blood and Marrow Transplant & Cellular Therapy, University of Minnesota, Minneapolis, MN 55455, USA. Electronic address: [email protected].

PMID: 34737067 DOI: 10.1016/j.ymthe.2021.10.026

Abstract

Disruption of CCR5 or CXCR4, the main human immunodeficiency virus type 1 (HIV-1) co-receptors, has been shown to protect primary human CD4

Copyright © 2021 Elsevier Inc. All rights reserved.

Keywords: Base editing; CCR5; CRISPR/Cas9; CXCR4; HIV; HSC

Conflict of interest statement

Declaration of interests D.R.L. is a consultant and equity owner of Beam Therapeutics, Prime Medicine, and Pairwise Plants, companies that use genome editing. D.R.L. and G.A.N. have filed patent appli

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