The Impact of Gene Editing and Gene Therapy on the Healthcare of Today
DOI:
https://doi.org/10.58445/rars.2710Keywords:
Gene editing, Genetic correctionAbstract
Advancements in gene editing and gene therapy have introduced a new era of possibilities in modern medicine. There are many gene editing methods, with CRISPR receiving most of the spotlight. Genetic correction shows promise for many different diseases and individuals and serves as a bridge between the medicine of today and the medicine of
tomorrow. With rapid progress, the lengthy clinical trial process and the emphasis on ethics ensure the safety of treatments and uphold the value of benefiting patients. The original thoughts on many diseases are evolving as these techniques advance. We are currently witnessing change in the field of medicine through the lens of genetics.
References
Aljabali, A. A. A., El-Tanani, M., & Tambuwala, M. M. (2024). Principles of CRISPR-Cas9
technology: Advancements in genome editing and emerging trends in Drug Delivery. Journal of
Drug Delivery Science and Technology, 92, 105338. https://doi.org/10.1016/j.jddst.2024.105338
Alkanli, S. S., Alkanli, N., Ay, A., & Albeniz, I. (2023). CRISPR/Cas9 Mediated Therapeutic
Approach in Huntington's Disease. Molecular neurobiology, 60(3), 1486–1498.
https://doi.org/10.1007/s12035-022-03150-5
American Society of Gene & Cell Therapy [ASGCT]. (2021, November 2). Gene editing.
https://patienteducation.asgct.org/gene-therapy-101/gene
editing#:~:text=What's%20the%20Difference%3A%20Gene%20Therapy,to%20treat%20or%20
prevent%20disease.
Ayanoğlu, F. B., Elçin, A. E., & Elçin, Y. M. (2020). Bioethical issues in genome editing by
CRISPR-Cas9 technology. Turkish journal of biology = Turk biyoloji dergisi, 44(2), 110–120.
https://doi.org/10.3906/biy-1912-52
Bierlaagh, M. C., Muilwijk, D., Beekman, J. M., & van der Ent, C. K. (2021). A new era for
people with cystic fibrosis. European journal of pediatrics, 180(9), 2731–2739.
https://doi.org/10.1007/s00431-021-04168-y
Carroll, D. (2021). A short, idiosyncratic history of genome editing. Gene and Genome Editing,
, 100002. https://doi.org/10.1016/j.ggedit.2021.100002
Fletcher, L. (2024, July 26). Back to basics - base & prime editing. Front Line Genomics.
https://frontlinegenomics.com/back-to-basics-base-and-prime-editing/
Fu, J., Ji, H. L., Naren, A. P., & Kirk, K. L. (2001). A cluster of negative charges at the amino
terminal tail of CFTR regulates ATP-dependent channel gating. The Journal of Physiology,
(Pt 2), 459–470. https://doi.org/10.1111/j.1469-7793.2001.0459c.xd
Hildebrandt, C. C., & Marron, J. M. (2018). Justice in CRISPR/Cas9 Research and Clinical
Applications. AMA journal of ethics, 20(9), E826–E833.
https://doi.org/10.1001/amajethics.2018.826
Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012). A
programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science
(New York, N.Y.), 337(6096), 816–821. https://doi.org/10.1126/science.1225829
Johns Hopkins Medicine. (n.d.). Genetic disorders.
https://www.hopkinsmedicine.org/health/genetic-disorders
Konstantakos, V., Nentidis, A., Krithara, A., & Paliouras, G. (2022). CRISPR-Cas9 gRNA
efficiency prediction: an overview of predictive tools and the role of deep learning. Nucleic acids
research, 50(7), 3616–3637. https://doi.org/10.1093/nar/gkac192
Lesté-Lasserre, C. (2024, June 13). Lung-targeted CRISPR therapy offers hope for cystic
fibrosis. New Scientist. https://www.newscientist.com/article/2435568-lung-targeted-crispr
therapy-offers-hope-for-cystic-fibrosis/
MedlinePlus. (2022, February 28). What are the ethical issues surrounding gene therapy?
https://medlineplus.gov/genetics/understanding/therapy/ethics/
National Human Genome Research Institute [NHGRI]. (2019, March 13). What are the ethical
concerns of genome editing? https://www.genome.gov/about-genomics/policy-issues/Genome
Editing/ethical-concerns
Razi Soofiyani, S., Baradaran, B., Lotfipour, F., Kazemi, T., & Mohammadnejad, L. (2013).
Gene therapy, early promises, subsequent problems, and recent breakthroughs. Advanced
Pharmaceutical Bulletin, 3(2), 249–255. https://doi.org/10.5681/apb.2013.041
Schleidgen, S., Dederer, H. G., Sgodda, S., Cravcisin, S., Lüneburg, L., Cantz, T., &
Heinemann, T. (2020). Human germline editing in the era of CRISPR-Cas: risk and uncertainty,
inter-generational responsibility, therapeutic legitimacy. BMC medical ethics, 21(1), 87.
https://doi.org/10.1186/s12910-020-00487-1
Sufian, S., & Garland-Thomson, R. (2024, February 20). The Dark Side of CRISPR. Scientific
American. https://www.scientificamerican.com/article/the-dark-side-of-crispr/
Varkey B. (2021). Principles of Clinical Ethics and Their Application to Practice. Medical
principles and practice: international journal of the Kuwait University, Health Science Centre,
(1), 17–28. https://doi.org/10.1159/000509119
Wang, C., Pan, C., Yong, H., Wang, F., Bo, T., Zhao, Y., Ma, B., He, W., & Li, M. (2023).
Emerging non-viral vectors for gene delivery. Journal of Nanobiotechnology, 21(1), 272.
https://doi.org/10.1186/s12951-023-02044-5
Yang, H., Ren, S., Yu, S., Pan, H., Li, T., Ge, S., Zhang, J., & Xia, N. (2020). Methods Favoring
Homology-Directed Repair Choice in Response to CRISPR/Cas9 Induced-Double Strand
Breaks. International journal of molecular sciences, 21(18), 6461.
Downloads
Posted
Categories
License
Copyright (c) 2025 Aarush Gupta
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.