CRISPR/Cas9 from bench to bedside: what clinicians need to know before application?
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LETTER TO THE EDITOR
Open Access
CRISPR/Cas9 from bench to bedside: what clinicians need to know before application? Zi-Qing Li1,2*
and Chao-Hong Li3
Abstract In October 2020, Dr. Emmanuelle Charpentier and Dr. Jennifer Doudna won the Nobel Prize in Chemistry for their pioneering work in precise genome editing using the CRISPR technology. Although CRISPR technology has developed rapidly in the last decade, there are still many uncertainties before eventual use in clinical settings. In this mini review, we summarize the current efforts in addressing the limitations of CRISPR technology and future directions. Keywords: CRISPR/Cas9, Nobel prize, Genome editing, Off-target effect, Ethical concerns Dear editor, In October 2020, the Nobel Committee announced the award of Nobel Prize in Chemistry to Dr. Emmanuelle Charpentier and Dr. Jennifer Doudna for their pioneering work in precise genome editing with the clustered regularly interspaced short palindromic repeats (CRISPR) technology. The basic features of CRISPR were first recognized by Japanese scientists in 1987 and officially termed as “CRISPR” in 2002, but it was not further developed until the early 2000s, when it was used as a powerful genome editing platform [1, 2]. This technology allows exceptionally precise genome editing in a wide range of species and extends our ability to investigate the contribution of genetic factors to various unexplained phenotypes and diseases. After the rapid development in laboratory settings, CRISPR technology has thunderingly reached the stage of applied biotechnology, and more—gene therapy. Meanwhile, the limitations of this technology, the unknown functions of candidate genes, and the ethical concerns of human use became increasingly emerging before clinical application [3]. * Correspondence: [email protected]; [email protected] 1 Department of Joint Surgery, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan 250021, China 2 Department of Basic and Translational Sciences, School of Dental Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA Full list of author information is available at the end of the article
In a previous issue of Military Medical Research, Prof. Xiao Yang [4, 5] provided an overview of CRISPR/Cas9mediated genome engineering and its current applications. In the same issue, Dr. Chun-xiao Li and Dr. Hai-li Qian expressed concerns about the limitations of this technology and ethical issues in future use [4, 5]. Indeed, it took only less than 10 years from the development of CRISPR/Cas9 as a basic science research tool to the translation of CRISPR technologies (CRISPR/Cas9-mediated genome editing, CRISPR activation, and CRISPR interference) into powerful therapeutic implement [3, 6]. Uncertainties still exist, and if we do not pay enough attention to evidence-based clinical standards and proceed rushly, there may be consequences that we cannot afford. In this mini-review, we summarize the current efforts in addressing the limitations of CRISPR technology and fu
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