CRISPR/Cas9 genome editing technology applications in biological and biomedical fields

2021 ◽  
Author(s):  
Moataz Dowaidar

Gene therapy is a way of mending or replacing a gene in an undesirable or non-functional cell. Although used in both animals and plants, gene therapy is most usually linked with humans. Because there are so many genetic disorders caused by genetic abnormalities or unwanted gene expression, gene therapy is promising to treat and even cure many diseases. The scientific and pharmaceutical sectors are becoming interested in gene therapy.CRISPR was initially detected in prokaryotic organisms, bacteria and archaea genomes. Although nucleotide sequences are regularly discovered in many bacteria and archaea, the scientific community has not realized its importance for over a decade. People used these diverse DNA sequences as a diagnostic for genotyping and therefore considered them as a distinctive feature for each particular microbe. Scientists are beginning to comprehend that the CRISPR/Cas system is a prokaryotic defense system's adaptive immunity to viruses, due to the discovery of CRISPR-associated protein (Cas) and the use of recombinant DNA technology. This recently discovered CRISPR/Cas system was swiftly developed as a tool for editing a specific gene in a genome. Since 2012, CRISPR/Cas9 genome editing technology has been quickly researched and applied in several biological and biomedical fields. For various basic and practical research reasons, as well as biotechnological applications in agriculture and healthcare, CRISPR/Cas9 technology has altered and improved greatly over the past five years. Base editor invention and prime editing technology by fusing a Cas endonuclease with other functional enzymes, such as base converter enzymes, is one of several milestones in this fast progress.

2020 ◽  
Vol 19 (16) ◽  
pp. 1912-1919
Author(s):  
Wenlou Liu ◽  
Chunsheng Yang ◽  
Yanqun Liu ◽  
Guan Jiang

Genome editing refers to changing the genome sequence of an organism by knockout, insertion, and site mutation, resulting in changes in the genetic information of the organism. The clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein-9 nuclease (Cas9) system is a genome editing technique developed by the acquired immune system in the microbes, such as bacteria and archaebacteria, which targets and edits genome sequences according to the principle of complementary base pairing. This technique can be used to edit endogenous genomic DNA sequences in organisms accurately and has been widely used in fields, such as biotechnology, cancer gene therapy, and dermatology. In this review, we summarize the history, structure, mechanism, and application of CRISPR/Cas9 in gene therapy and dermatological diseases.


2016 ◽  
Vol 44 (3) ◽  
pp. 160-164
Author(s):  
Khadija Akther Jhuma ◽  
ASM Giasuddin ◽  
AM Mujibul Haq ◽  
Shahryar Nabi

In 1950s, double-stranded deoxyribonucleic acid (DNA) had been identified as the biochemical basis of heredity and ‘gene’ had been shown to be a segment of DNA. Accordingly, vast majority of hereditary disorders involve changes, i.e. mutations in specific genes. Can medical treatment leading to genetic cure of these hereditary disorders possible? The answer is ‘yes’ as the initial thoughts of ‘Gene therapy’ have been transformed into reality in recent times. Gene therapy is the application of various technologies including ‘Recombinant DNA Technology’ for introduction of a relevant functional gene, i.e. exogenous DNA, into a cell to achieve therapeutic effects for genetic disorders. Since 1990, gene therapy has become standard treatment for a number of primary immunodeficiency diseases (PIDs) such as adenosine deaminase deficiency form of severe combined immunodeficiency (ADA-SCID), SCID-X1, Wiskott-Aldrich syndrome (WAS), chronic granulomatous disease (CGD) and others. As we eagerly wait until the results of ongoing further clinical trials are available, updated accounts of the status of ‘Gene Therapy’ for selective PIDs are presented in this review article.Bangladesh Med J. 2015 Sep; 44 (3): 160-164


2017 ◽  
Vol 37 (01) ◽  
pp. 45-52 ◽  
Author(s):  
Simone Haas ◽  
Viviane Dettmer ◽  
Toni Cathomen

SummaryTargeted genome editing with designer nucleases, such as zinc finger nucleases, TALE nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic disorders, which have not been amenable to conventional gene-addition-type gene therapy approaches, such as disorders with dominant inheritance or diseases caused by mutations in tightly regulated genes, can now be treated by precise genome surgery. Moreover, engineered nucleases enable novel genetic interventions to fight infectious diseases or to improve cancer immunotherapies. Here, we review the development of the different classes of programmable nucleases, discuss the challenges and improvements in translating gene editing into clinical use, and give an outlook on what applications can expect to enter the clinic in the near future.


Recombinant DNA technology has contributed greatly to the precision of chromosome analysis in man. Breakpoints of chromosome deletions and rearrangements may be defined on a chromosome map whose landmarks are the loci of DNA sequences rather than Giemsa bands. Flow cytogenetics allows the extent of chromosome duplications and deletions to be measured more precisely than has hitherto been possible. DNA probes can reveal hidden translocations through the application of in situ hybridization, and may be used as markers to determine the parental origin of non-disjunction. It is evident that a study of the pathology of human chromosomes now requires the combined skills of recombinant DNA and cytology.


2019 ◽  
Vol 63 (4) ◽  
pp. 457-468 ◽  
Author(s):  
Mark A. J. Roberts

Abstract DNA present in all our cells acts as a template by which cells are built. The human genome project, reading the code of the DNA within our cells, completed in 2003, is undoubtedly one of the great achievements of modern bioscience. Our ability to achieve this and to further understand and manipulate DNA has been tightly linked to our understanding of the bacterial and viral world. Outside of the science, the ability to understand and manipulate this code has far-reaching implications for society. In this article, we explore some of the basic techniques that enable us to read, copy and manipulate DNA sequences alongside a brief consideration of some of the implications for society.


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