Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation

CRISPR offers new hope for many patients and promises to transform the way we think of future therapies. Ensuring safety of CRISPR therapeutics is a top priority for clinical translation and specific recommendations have been recently released by the FDA. Rapid progress in the preclinical and clinic...

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Main Authors: Radwa Ewaisha, Karen S. Anderson
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-02-01
Series:Frontiers in Bioengineering and Biotechnology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fbioe.2023.1138596/full
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author Radwa Ewaisha
Radwa Ewaisha
Karen S. Anderson
author_facet Radwa Ewaisha
Radwa Ewaisha
Karen S. Anderson
author_sort Radwa Ewaisha
collection DOAJ
description CRISPR offers new hope for many patients and promises to transform the way we think of future therapies. Ensuring safety of CRISPR therapeutics is a top priority for clinical translation and specific recommendations have been recently released by the FDA. Rapid progress in the preclinical and clinical development of CRISPR therapeutics leverages years of experience with gene therapy successes and failures. Adverse events due to immunogenicity have been a major setback that has impacted the field of gene therapy. As several in vivo CRISPR clinical trials make progress, the challenge of immunogenicity remains a significant roadblock to the clinical availability and utility of CRISPR therapeutics. In this review, we examine what is currently known about the immunogenicity of CRISPR therapeutics and discuss several considerations to mitigate immunogenicity for the design of safe and clinically translatable CRISPR therapeutics.
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spelling doaj.art-3c47b5197cda478496567a64a50b5d482023-02-16T08:04:41ZengFrontiers Media S.A.Frontiers in Bioengineering and Biotechnology2296-41852023-02-011110.3389/fbioe.2023.11385961138596Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translationRadwa Ewaisha0Radwa Ewaisha1Karen S. Anderson2Department of Microbiology and Immunology, Faculty of Pharmacy, Alexandria University, Alexandria, EgyptDepartment of Microbiology and Immunology, School of Pharmacy, Newgiza University, Newgiza, EgyptCenter for Personalized Diagnostics, Biodesign Institute, Arizona State University, Tempe, AZ, United StatesCRISPR offers new hope for many patients and promises to transform the way we think of future therapies. Ensuring safety of CRISPR therapeutics is a top priority for clinical translation and specific recommendations have been recently released by the FDA. Rapid progress in the preclinical and clinical development of CRISPR therapeutics leverages years of experience with gene therapy successes and failures. Adverse events due to immunogenicity have been a major setback that has impacted the field of gene therapy. As several in vivo CRISPR clinical trials make progress, the challenge of immunogenicity remains a significant roadblock to the clinical availability and utility of CRISPR therapeutics. In this review, we examine what is currently known about the immunogenicity of CRISPR therapeutics and discuss several considerations to mitigate immunogenicity for the design of safe and clinically translatable CRISPR therapeutics.https://www.frontiersin.org/articles/10.3389/fbioe.2023.1138596/fullCRISPR-Casimmunogenicityclinical translationCas9genome editinggene therapy
spellingShingle Radwa Ewaisha
Radwa Ewaisha
Karen S. Anderson
Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
Frontiers in Bioengineering and Biotechnology
CRISPR-Cas
immunogenicity
clinical translation
Cas9
genome editing
gene therapy
title Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
title_full Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
title_fullStr Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
title_full_unstemmed Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
title_short Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
title_sort immunogenicity of crispr therapeutics critical considerations for clinical translation
topic CRISPR-Cas
immunogenicity
clinical translation
Cas9
genome editing
gene therapy
url https://www.frontiersin.org/articles/10.3389/fbioe.2023.1138596/full
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