Challenges and Opportunities in the Application of CRISPR-Cas9: A Review on Genomic Editing and Therapeutic Potentials

• Published in: Medical principles and practice pp. 1 - 30
• Main Authors: Karimi, Mohammad Ali, Paryan, Mahdiesadat, Behrouzian Fard, Ghazaleh, Sadeghian, Hamid, Zarrinfar, Hossein, Hosseini Bafghi, Mahdi
• Format: Journal Article
• Language: English
• Published: Switzerland 17.07.2025
• Subjects: Off-target effects; Genetic engineering; Gene therapy; CRISPR-Cas9; Gene editing
• ISSN: 1011-7571; 1423-0151; 1423-0151
• DOI: 10.1159/000547334

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and the CRISPR-associated protein 9 (Cas9) constitute a revolutionary gene-editing technology, allowing precise DNA modifications with vast potential for disease treatment and the creation of genetically modified organisms. This system consists of various components designed to target specific genes, requiring efficient nuclear access within target cells through diverse delivery methods, including physical techniques and carrier-based approaches. Despite its transformative promise, CRISPR faces several challenges, such as efficient cellular delivery, off-target effects, immune responses, optimization of editing efficiency, and ethical concerns. Overcoming these hurdles is crucial for fully harnessing its capabilities. However, CRISPR-Cas9 offers remarkable opportunities for pioneering gene therapies across various disorders, including cancer, and could revolutionize agriculture by engineering disease-resistant crops. A key strength of CRISPR lies in its adaptability to a broad spectrum of genes, significantly enhancing its versatility. The technology's potential further expands through emerging interdisciplinary integrations, such as artificial intelligence (AI), machine learning, and biological imaging. These advancements can refine CRISPR’s precision, improve efficiency, and mitigate existing limitations, positioning it as an indispensable tool in future genetic research. Overall, CRISPR-Cas9 promises transformative progress in healthcare and agriculture, solidifying its role as a cornerstone in the evolution of genetic engineering.