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CRISPR library screening to develop HEK293-derived cell lines with improved lentiviral vector titers

Lentiviral (LV) vectors have emerged as powerful tools for treating genetic and acquired human diseases. As clinical studies and commercial demands have progressed, there has been a growing need for large amounts of purified LV vectors. To help meet this demand, we developed CRISPR library screening...

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Autores principales: Iaffaldano, Brian J., Marino, Michael P., Reiser, Jakob
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10373892/
https://www.ncbi.nlm.nih.gov/pubmed/37520398
http://dx.doi.org/10.3389/fgeed.2023.1218328
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author Iaffaldano, Brian J.
Marino, Michael P.
Reiser, Jakob
author_facet Iaffaldano, Brian J.
Marino, Michael P.
Reiser, Jakob
author_sort Iaffaldano, Brian J.
collection PubMed
description Lentiviral (LV) vectors have emerged as powerful tools for treating genetic and acquired human diseases. As clinical studies and commercial demands have progressed, there has been a growing need for large amounts of purified LV vectors. To help meet this demand, we developed CRISPR library screening methods to identify genetic perturbations in human embryonic kidney 293 (HEK293) cells and their derivatives that may increase LV vector titers. Briefly, LV vector-based Human CRISPR Activation and Knockout libraries (Calabrese and Brunello) were used to modify HEK293 and HEK293T cells. These cell populations were then expanded, and integrated LV vector genomes were rescued by transfection. LV vectors were harvested, and the process of sequential transduction and rescue-transfection was iterated. Through this workflow, guide RNAs (gRNAs) that target genes that may suppress or enhance LV vector production were enriched and identified with Next-Generation Sequencing (NGS). Though more work is needed to test genes identified in this screen, we expect that perturbations of genes we identified here, such as TTLL12, which is an inhibitor of antiviral innate immunity may be introduced and multiplexed to yield cell lines with improved LV vector productivity.
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spelling pubmed-103738922023-07-28 CRISPR library screening to develop HEK293-derived cell lines with improved lentiviral vector titers Iaffaldano, Brian J. Marino, Michael P. Reiser, Jakob Front Genome Ed Genome Editing Lentiviral (LV) vectors have emerged as powerful tools for treating genetic and acquired human diseases. As clinical studies and commercial demands have progressed, there has been a growing need for large amounts of purified LV vectors. To help meet this demand, we developed CRISPR library screening methods to identify genetic perturbations in human embryonic kidney 293 (HEK293) cells and their derivatives that may increase LV vector titers. Briefly, LV vector-based Human CRISPR Activation and Knockout libraries (Calabrese and Brunello) were used to modify HEK293 and HEK293T cells. These cell populations were then expanded, and integrated LV vector genomes were rescued by transfection. LV vectors were harvested, and the process of sequential transduction and rescue-transfection was iterated. Through this workflow, guide RNAs (gRNAs) that target genes that may suppress or enhance LV vector production were enriched and identified with Next-Generation Sequencing (NGS). Though more work is needed to test genes identified in this screen, we expect that perturbations of genes we identified here, such as TTLL12, which is an inhibitor of antiviral innate immunity may be introduced and multiplexed to yield cell lines with improved LV vector productivity. Frontiers Media S.A. 2023-07-13 /pmc/articles/PMC10373892/ /pubmed/37520398 http://dx.doi.org/10.3389/fgeed.2023.1218328 Text en Copyright © 2023 Iaffaldano, Marino and Reiser. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Genome Editing
Iaffaldano, Brian J.
Marino, Michael P.
Reiser, Jakob
CRISPR library screening to develop HEK293-derived cell lines with improved lentiviral vector titers
title CRISPR library screening to develop HEK293-derived cell lines with improved lentiviral vector titers
title_full CRISPR library screening to develop HEK293-derived cell lines with improved lentiviral vector titers
title_fullStr CRISPR library screening to develop HEK293-derived cell lines with improved lentiviral vector titers
title_full_unstemmed CRISPR library screening to develop HEK293-derived cell lines with improved lentiviral vector titers
title_short CRISPR library screening to develop HEK293-derived cell lines with improved lentiviral vector titers
title_sort crispr library screening to develop hek293-derived cell lines with improved lentiviral vector titers
topic Genome Editing
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10373892/
https://www.ncbi.nlm.nih.gov/pubmed/37520398
http://dx.doi.org/10.3389/fgeed.2023.1218328
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