Abstract
CRISPR-Cas systems, widespread in bacteria and archaea, are mainly responsible for adaptive cellular immunity against exogenous DNA (plasmid and phage). However, the latest research shows their involvement in other functions, such as gene expression regulation, DNA repair and virulence. In recent years, they have undergone intensive research as convenient tools for genomic editing, with Cas9 being the most commonly used nuclease. Gene editing may be of interest in biotechnology, medicine (treatment of inherited disorders, cancer, etc.), and in the development of model systems for various genetic diseases. The dCas9 system, based on a modified Cas9 devoid of nuclease activity, called CRISPRi, is widely used to control gene expression in bacteria for new drug biotargets validation and is also promising for therapy of genetic diseases. In addition to direct use for genomic editing in medicine, CRISPR-Cas can also be used in diagnostics, for microorganisms’ genotyping, controlling the spread of drug resistance, or even directly as “smart” antibiotics. This review focuses on the main applications of CRISPR-Cas in medicine, and challenges and perspectives of these approaches.
Highlights
Many human diseases are genetically determined: as of today, over 6000 hereditary diseases are known to be caused by gene and chromosomal DNA mutations, both nuclear and mitochondrial [1].The treatment of hereditary diseases has been mostly symptomatic, until recently gene therapy has emerged as a fundamentally new approach, aimed at eliminating directly the cause of the disease by correcting mutations
They usually consist of two parts: the Clustered regularly interspaced short palindromic repeats (CRISPR) cassette, which consists of a number of unique sequences of about the same length, separated by short repeating segments, and a locus of CRISPR-associated genes
CRISPRi systems, based on dCas9 protein depleted of nuclease activity are currently widely used for prokaryotic genes’ functional studies and identification of novel metabolic pathways for new biotargets identification
Summary
Many human diseases are genetically determined: as of today, over 6000 hereditary diseases are known to be caused by gene and chromosomal DNA mutations, both nuclear and mitochondrial [1]. Sci. 2020, 10, 9001 since the DR set usually differed within one species It was only in the 2000s when the role of CRISPR-Cas systems in the bacterial immunity was described [5,6]. These systems were well studied subsequently, with other functions, such as gene expression regulation, DNA repair, and even indirect participation in the process of host infection, described for them aside from providing adaptive immune response [7,8,9,10,11,12]. These studies include, but are not limited to: genotyping and epidemiological studies, studying the role of individual genes in the pathogenesis and survival of bacteria (including search for new drugs’ biotargets), and for targeted killing of certain bacterial strains, including drug resistant ones
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
