CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future
A series of recent discoveries harnessing the adaptive immune system of prokaryotes to perform targeted genome editing is having a transformative influence across the biological sciences. The discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas)...
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Published in | Frontiers in oncology Vol. 10; p. 1387 |
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Main Authors | , , |
Format | Journal Article |
Language | English |
Published |
Switzerland
Frontiers Media S.A
07.08.2020
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Subjects | |
Online Access | Get full text |
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Summary: | A series of recent discoveries harnessing the adaptive immune system of prokaryotes to perform targeted genome editing is having a transformative influence across the biological sciences. The discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) proteins has expanded the applications of genetic research in thousands of laboratories across the globe and is redefining our approach to gene therapy. Traditional gene therapy has raised some concerns, as its reliance on viral vector delivery of therapeutic transgenes can cause both insertional oncogenesis and immunogenic toxicity. While viral vectors remain a key delivery vehicle, CRISPR technology provides a relatively simple and efficient alternative for site-specific gene editing, obliviating some concerns raised by traditional gene therapy. Although it has apparent advantages, CRISPR/Cas9 brings its own set of limitations which must be addressed for safe and efficient clinical translation. This review focuses on the evolution of gene therapy and the role of CRISPR in shifting the gene therapy paradigm. We review the emerging data of recent gene therapy trials and consider the best strategy to move forward with this powerful but still relatively new technology. |
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Bibliography: | ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-3 content type line 23 ObjectType-Review-1 Lead Contact This article was submitted to Cancer Genetics, a section of the journal Frontiers in Oncology Edited by: Israel Gomy, Dana–Farber Cancer Institute, United States Reviewed by: Nan Wu, Peking Union Medical College Hospital (CAMS), China; Tanja Kunej, University of Ljubljana, Slovenia; Martin Roffe, A.C. Camargo Cancer Center, Brazil |
ISSN: | 2234-943X 2234-943X |
DOI: | 10.3389/fonc.2020.01387 |