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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Bibliographic Details
Published in:Frontiers in bioengineering and biotechnology 2023-02, Vol.11, p.1138596
Main Authors: Ewaisha, Radwa, Anderson, Karen S
Format: Article
Language:English
Subjects:
Bioengineering and Biotechnology
Cas9
clinical translation
CRISPR-Cas
gene therapy
genome editing
immunogenicity
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Summary: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 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.
ISSN:2296-4185
2296-4185
DOI:10.3389/fbioe.2023.1138596