CRISPR technology demonstrates potential in targeting monogenic and polygenic disorders, suggesting new gene therapies.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has emerged as a groundbreaking tool for genome editing, revolutionizing the management of genetic diseases. Its precision, efficiency, and adaptability enable targeted modification of DNA sequences, offering therapeutic potential for a wide range of inherited disorders. This technology utilizes the Cas9 endonuclease, guided by RNA, to introduce site-specific cuts in the genome, facilitating the correction, disruption, or replacement of genes. Applications include addressing monogenic disorders such as cystic fibrosis, sickle cell anemia, and Duchenne muscular dystrophy, as well as polygenic conditions and diseases linked to somatic mutations, including certain cancers. Furthermore, CRISPR has opened avenues for ex vivo and in vivo gene therapies, including the use of stem cells and viral vectors for effective delivery. While significant ethical and technical challenges remain, such as the risk of unintended genetic modifications and equitable access to therapy, CRISPR represents a paradigm shift in the treatment of genetic diseases. These abstract highlights the current state, challenges, and prospects of CRISPR technology as a transformative tool in precision medicine.
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Ogunlakin et al. (2025) studied this question.
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