CRISPR AND GENE EDITING: REVOLUTIONIZING TREATMENT OF GENETIC DISORDERS
Keywords:
CRISPR, gene editing, genetic disorders, Cas9, personalized medicine, regenerative medicine, molecular biology, biotechnology, ethical considerations, clinical applications.Abstract
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) and gene editing technologies have emerged as groundbreaking tools in modern biomedical research, offering unprecedented precision in modifying genetic material. These technologies enable targeted correction of disease-causing mutations, paving the way for the treatment of previously incurable genetic disorders such as cystic fibrosis, sickle cell anemia, and Duchenne muscular dystrophy. This article explores the molecular mechanisms of CRISPR-Cas9, recent advancements in gene editing approaches, and their clinical applications. Furthermore, it highlights ethical considerations, safety challenges, and the potential of these technologies to transform personalized medicine. The findings suggest that CRISPR and gene editing not only offer therapeutic hope for patients with genetic disorders but also represent a paradigm shift in the field of regenerative medicine and biomedical innovation.
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References
Doudna, J. A., & Charpentier, E. (2014). The new frontier of genome engineering with CRISPR-Cas9. Science, 346(6213), 1258096.
Hsu, P. D., Lander, E. S., & Zhang, F. (2014). Development and applications of CRISPR-Cas9 for genome engineering. Cell, 157(6), 1262–1278.
Komor, A. C., Badran, A. H., & Liu, D. R. (2017). CRISPR-based technologies for the manipulation of eukaryotic genomes. Cell, 169(3), 559–575.
Anzalone, A. V., Randolph, P. B., Davis, J. R., et al. (2019). Search-and-replace genome editing without double-strand breaks or donor DNA. Nature, 576, 149–157.
Cox, D. B., Platt, R. J., & Zhang, F. (2015). Therapeutic genome editing: prospects and challenges. Nature Medicine, 21, 121–131.
Ma, H., Marti-Gutierrez, N., Park, S. W., et al. (2017). Correction of a pathogenic gene mutation in human embryos. Nature, 548, 413–419.
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