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Era of Genomic Medicine: A Narrative Review on CRISPR Technology as a Potential Therapeutic Tool for Human Diseases
Abstract
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) provides acquired immunity in microorganisms against exogenous DNA that may hinder the survival of the organism. Pioneering work by Doudna and Charpentier in 2012 resulted in the creation of the CRISPR/Cas9 genome editing tool on the basis of this concept. The aim of this was to create a rapid, efficient, and versatile genome-editing tool to facilitate genetic manipulation. The mechanism relies on two components: the RNA guide which acts as a sentinel and a Cas protein complex which functions as a highly precise molecular knife. The guide RNA can be modified to match a DNA sequence of interest in the cell and accordingly be used to rectify mutations that may otherwise cause disease. Within a few years following the development of the CRISPR/Cas9 tool, its usage has become ubiquitous. Its influence extends into many fields of biological sciences from biotechnology and biochemistry to molecular biology and biomedical sciences. The following review aims at shedding some light on to the applications of the CRISPR/Cas9 tool in the field of biomedical sciences, particularly gene therapy. An insight with relation to a few of the many diseases that are being tackled with the aid of the CRISPR/Cas9 mechanism and the trends, successes, and challenges of this application as a gene therapy are discussed in this review.
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“Immunogenicity, lack of a safe and efficient delivery system to the target, off-target effect, and ethical issues have been the major barriers to extend the technology in clinical applications.52”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [52]
In the citing paper
“Since the technology is still in its infancy and knowledge about the genome is limited, many scientists restrain that it still needs a lot of work to increase its accuracy and make sure that changes made in one part of the genome do not have unforeseen consequences, especially in the application towards human trials.52”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [52]
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“Furthermore, CRISPR/Cas-9 gene editing has been challenged by ethics and safety all over the world.”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [52]
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Cited by
Sorted by citations
Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing
Mengstie, M. A., & Wondimu, B. Z. (2021) · Biologics: Targets and Therapy · doi:10.2147/btt.s326422 · Cited by 478
Recent advancements in reducing the off-target effect of CRISPR/Cas9 genome editing
Asmamaw Mengstie, M., Teshome Azezew, M., Asmamaw Dejenie, T., Teshome, A. A., Tadele Admasu, F., Behaile Teklemariam, A., Tilahun Mulu, A., Mekonnen Agidew, M., Adugna, D. G., Geremew, H., et al. (2024) · Biologics: Targets and Therapy · doi:10.2147/btt.s429411 · Cited by 91
Metadata
- Authors
- Kotagama, O. W., Jayasinghe, C. D., & Abeysinghe, T.
- Affiliations
- Department of Chemistry, Faculty of Natural Sciences, The Open University of Sri Lanka, Nawala, Nugegoda, Sri Lanka
- Department of Zoology, Faculty of Natural Sciences, The Open University of Sri Lanka, Nawala, Nugegoda, Sri Lanka
- Venue
- BioMed Research International
- Publisher
- Wiley
- Published
- 2019-10-07
- Volume
- 2019
- Pages
- 1-15
- ISSN
- 2314-6133
- 2314-6141
- PMC
- PMC6800964
- Cited by
- 48
- References
- 9
- Language
- en
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