The recent emergence of genetic defects has led to the innovation of various genetic technique tools such as CRISPR-Cas9(Regularly interspaced short palindromic repeats), a recently revolutionary technique tool. It is a revolutionary technique that allows scientists to edit genes with exceptional accuracy, thus offering enormous potential for treating various genetic defects. It is a revolutionary technique that can be a game-changer in gene therapy (Janik et al., 2020). The context below discusses CRISPR-Cas9 and explains more research on how it works, how it could be applied to treat the genetic disorder and the potential risks of using this genetic technique.
There are various reasons why the revolutionary technique is an exciting tool in gene therapy, including the method, ongoing research, and the ability to edit genes directly. The versatility of the technique is essential as it is practical for an extensive range of diseases caused by genetic defects. Ongoing research is where researchers are constantly improving research for the efficiency of the technique, making it a promising tool for future gene therapy, as it is still in the early stages (Vaghari-Tabari et al., 2022). The ability of this technique to edit genes directly is another exciting feature, as it allows scientists to edit genes directly within an organism’s cell. The research shows that the CRISP-Cas9 technique is a future gene therapy application.
There are various ways in which the CRIPR-Cas9 revolutionary tool works as a tool for gene therapy. It is a two-component system inspired by a bacterial defence apparatus. The first component is a molecule called Cas9, a protein that acts as molecular scissors. The second component is a guide RNA (guiding ribonucleic acid), which acts as a GPS, directing Cas9 to the precise location in the genome that needs editing. The CRISP-Cas9 technique works by cellular repair, targeting and putting Cas9 in action. In detail, the cellular repair technique enables the cell’s natural repair machinery to kick in to mend the break. The cell repair process can be done in two ways. They include non-homologous end joining, which introduces small insertions in the cleavage site, which disrupts the targeted gene (Vaghari-Tabari et al., 2022). Homology-directed repair is the other way a cell’s natural repair machinery kicks in to mend the break. The CRISP-Cas9 technique uses a specialized Cas9 enzyme coupled with an engineered molecule that guides it to the target location and directly converts one DNA base.
The CRISPR-Cas9 technique tool has various applications that display the various genetic diseases it deals with. Some diseases include sickle cell disease, cystic fibrosis, beta-thalassemia and cancers (Janik et al., 2020). A description example includes sickle cell disease, where a single mutation in the haemoglobin gene disrupts red blood cell function, which could be treated by converting the faulty base back to the healthy form.
However, various potential risks of using the CRISP-Cas9 technique demand careful consideration. Some dangers include mosaicism, unintended immune response, and target effects. In detail, some cells have the desired correction in mosaicism, while others do not. This could lead to incomplete therapeutic benefits or unpredictable effects since gene therapy edits the population of cells. At the same time, this technique might only affect some cells uniformly. The method could trigger an unintended immune response, potentially reducing the effectiveness of the therapy. The potential risk of off-target risks is Cas9 cleaving unintended locations in the genome due to imperfect guide RNA targeting, which could lead to unintended consequences (Vaghari-Tabari et al., 2022). CRISPR-Cas9 represents a powerful tool with the potential to revolutionize medicine, and it offers a glimpse of future solutions to deal with genetic diseases.
References
Janik, E., Niemcewicz, M., Ceremuga, M., Krzowski, L., Saluk-Bijak, J., & Bijak, M. (2020). Various aspects of a gene editing system—CRISPR–cas9. International Journal of Molecular Sciences, 21(24), 9604.
Vaghari-Tabari, M., Hassanpour, P., Sadeghsoltani, F., Malakoti, F., Alemi, F., Qujeq, D., … & Yousefi, B. (2022). CRISPR/Cas9 gene editing: a new approach for overcoming drug resistance in cancer. Cellular & Molecular Biology Letters, 27(1), 49.
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