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Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes

CRISPR-mediated genome editing of primary human lymphocytes is typically carried out via electroporation, which can be cytotoxic, cumbersome and costly. Here we show that the yields of edited primary human lymphocytes can be increased substantially by delivering a CRISPR ribonucleoprotein mixed with...

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Autores principales: Foss, Dana V., Muldoon, Joseph J., Nguyen, David N., Carr, Daniel, Sahu, Srishti U., Hunsinger, John M., Wyman, Stacia K., Krishnappa, Netravathi, Mendonsa, Rima, Schanzer, Elaine V., Shy, Brian R., Vykunta, Vivasvan S., Allain, Vincent, Li, Zhongmei, Marson, Alexander, Eyquem, Justin, Wilson, Ross C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10129304/
https://www.ncbi.nlm.nih.gov/pubmed/37147433
http://dx.doi.org/10.1038/s41551-023-01032-2
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author Foss, Dana V.
Muldoon, Joseph J.
Nguyen, David N.
Carr, Daniel
Sahu, Srishti U.
Hunsinger, John M.
Wyman, Stacia K.
Krishnappa, Netravathi
Mendonsa, Rima
Schanzer, Elaine V.
Shy, Brian R.
Vykunta, Vivasvan S.
Allain, Vincent
Li, Zhongmei
Marson, Alexander
Eyquem, Justin
Wilson, Ross C.
author_facet Foss, Dana V.
Muldoon, Joseph J.
Nguyen, David N.
Carr, Daniel
Sahu, Srishti U.
Hunsinger, John M.
Wyman, Stacia K.
Krishnappa, Netravathi
Mendonsa, Rima
Schanzer, Elaine V.
Shy, Brian R.
Vykunta, Vivasvan S.
Allain, Vincent
Li, Zhongmei
Marson, Alexander
Eyquem, Justin
Wilson, Ross C.
author_sort Foss, Dana V.
collection PubMed
description CRISPR-mediated genome editing of primary human lymphocytes is typically carried out via electroporation, which can be cytotoxic, cumbersome and costly. Here we show that the yields of edited primary human lymphocytes can be increased substantially by delivering a CRISPR ribonucleoprotein mixed with an amphiphilic peptide identified through screening. We evaluated the performance of this simple delivery method by knocking out genes in T cells, B cells and natural killer cells via the delivery of Cas9 or Cas12a ribonucleoproteins or an adenine base editor. We also show that peptide-mediated ribonucleoprotein delivery paired with an adeno-associated-virus-mediated homology-directed repair template can introduce a chimaeric antigen receptor gene at the T-cell receptor α constant locus, and that the engineered cells display antitumour potency in mice. The method is minimally perturbative, does not require dedicated hardware, and is compatible with multiplexed editing via sequential delivery, which minimizes the risk of genotoxicity. The peptide-mediated intracellular delivery of ribonucleoproteins may facilitate the manufacturing of engineered T cells.
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spelling pubmed-101293042023-04-27 Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes Foss, Dana V. Muldoon, Joseph J. Nguyen, David N. Carr, Daniel Sahu, Srishti U. Hunsinger, John M. Wyman, Stacia K. Krishnappa, Netravathi Mendonsa, Rima Schanzer, Elaine V. Shy, Brian R. Vykunta, Vivasvan S. Allain, Vincent Li, Zhongmei Marson, Alexander Eyquem, Justin Wilson, Ross C. Nat Biomed Eng Article CRISPR-mediated genome editing of primary human lymphocytes is typically carried out via electroporation, which can be cytotoxic, cumbersome and costly. Here we show that the yields of edited primary human lymphocytes can be increased substantially by delivering a CRISPR ribonucleoprotein mixed with an amphiphilic peptide identified through screening. We evaluated the performance of this simple delivery method by knocking out genes in T cells, B cells and natural killer cells via the delivery of Cas9 or Cas12a ribonucleoproteins or an adenine base editor. We also show that peptide-mediated ribonucleoprotein delivery paired with an adeno-associated-virus-mediated homology-directed repair template can introduce a chimaeric antigen receptor gene at the T-cell receptor α constant locus, and that the engineered cells display antitumour potency in mice. The method is minimally perturbative, does not require dedicated hardware, and is compatible with multiplexed editing via sequential delivery, which minimizes the risk of genotoxicity. The peptide-mediated intracellular delivery of ribonucleoproteins may facilitate the manufacturing of engineered T cells. Nature Publishing Group UK 2023-04-25 2023 /pmc/articles/PMC10129304/ /pubmed/37147433 http://dx.doi.org/10.1038/s41551-023-01032-2 Text en © The Author(s), under exclusive licence to Springer Nature Limited 2023, Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law. This article is made available via the PMC Open Access Subset for unrestricted research re-use and secondary analysis in any form or by any means with acknowledgement of the original source. These permissions are granted for the duration of the World Health Organization (WHO) declaration of COVID-19 as a global pandemic.
spellingShingle Article
Foss, Dana V.
Muldoon, Joseph J.
Nguyen, David N.
Carr, Daniel
Sahu, Srishti U.
Hunsinger, John M.
Wyman, Stacia K.
Krishnappa, Netravathi
Mendonsa, Rima
Schanzer, Elaine V.
Shy, Brian R.
Vykunta, Vivasvan S.
Allain, Vincent
Li, Zhongmei
Marson, Alexander
Eyquem, Justin
Wilson, Ross C.
Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes
title Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes
title_full Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes
title_fullStr Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes
title_full_unstemmed Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes
title_short Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes
title_sort peptide-mediated delivery of crispr enzymes for the efficient editing of primary human lymphocytes
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10129304/
https://www.ncbi.nlm.nih.gov/pubmed/37147433
http://dx.doi.org/10.1038/s41551-023-01032-2
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