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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Veröffentlicht in:Frontiers in oncology Jg. 10; S. 1387
Hauptverfasser: Uddin, Fathema, Rudin, Charles M., Sen, Triparna
Format: Journal Article
Sprache:Englisch
Veröffentlicht: Switzerland Frontiers Media S.A 07.08.2020
Schlagworte:
clinical trial
CRISPR/Cas9
ethics
gene therapy
homology-directed repair (HDR)
non-homologous end joining (NHEJ)
Oncology
clinical trial
gene therapy
homology-directed repair (HDR)
CRISPR/Cas9
ethics
non-homologous end joining (NHEJ)
ISSN:2234-943X, 2234-943X
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Zusammenfassung: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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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