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CRISPR/Cas9 for Cancer Therapy: Hopes and Challenges

1
Molecular Cytogenetics Group, Human Cancer Genetics Program, Centro Nacional de Investigaciones Oncológicas (CNIO), 28029 Madrid, Spain
2
Josep Carreras Leukemia Research Institute and Department of Biomedicine, School of Medicine, University of Barcelona, 08036 Barcelona, Spain
3
Instituciò Catalana de Recerca i Estudis Avançats (ICREA), 08010 Barcelona, Spain
4
Centro de Investigación Biomédica en Red de Cáncer (CIBER-ONC), ISCIII, 08010 Barcelona, Spain
*
Authors to whom correspondence should be addressed.
These authors contributed equally to this work.
Biomedicines 2018, 6(4), 105; https://doi.org/10.3390/biomedicines6040105
Received: 17 October 2018 / Revised: 2 November 2018 / Accepted: 5 November 2018 / Published: 12 November 2018
(This article belongs to the Special Issue Stem Cells and Cancer Therapeutics)
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Abstract

Cancer is the second leading cause of death globally and remains a major economic and social burden. Although our understanding of cancer at the molecular level continues to improve, more effort is needed to develop new therapeutic tools and approaches exploiting these advances. Because of its high efficiency and accuracy, the CRISPR-Cas9 genome editing technique has recently emerged as a potentially powerful tool in the arsenal of cancer therapy. Among its many applications, CRISPR-Cas9 has shown an unprecedented clinical potential to discover novel targets for cancer therapy and to dissect chemical-genetic interactions, providing insight into how tumours respond to drug treatment. Moreover, CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications. Perhaps more importantly, the ability of CRISPR-Cas9 to accurately edit genes, not only in cell culture models and model organisms but also in humans, allows its use in therapeutic explorations. In this review, we discuss important considerations for the use of CRISPR/Cas9 in therapeutic settings and major challenges that will need to be addressed prior to its clinical translation for a complex and polygenic disease such as cancer. View Full-Text
Keywords: CRISPR; cancer models; genome engineering; Cas9; advance therapy CRISPR; cancer models; genome engineering; Cas9; advance therapy
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Martinez-Lage, M.; Puig-Serra, P.; Menendez, P.; Torres-Ruiz, R.; Rodriguez-Perales, S. CRISPR/Cas9 for Cancer Therapy: Hopes and Challenges. Biomedicines 2018, 6, 105.

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