<p class="first" id="d758106e113">At present, the idea of genome modification has
revolutionized the modern therapeutic
research era. Genome modification studies have traveled a long way from gene modifications
in primary cells to genetic modifications in animals. The targeted genetic modification
may result in the modulation (i.e., either upregulation or downregulation) of the
predefined gene expression. Clustered regularly interspaced short palindromic repeats
(CRISPR)-CRISPR-associated nuclease 9 (Cas9) is a promising genome-editing tool that
has therapeutic potential against incurable genetic disorders by modifying their DNA
sequences. In comparison with other genome-editing techniques, CRISPR-Cas9 is simple,
efficient, and very specific. This enabled CRISPR-Cas9 genome-editing technology to
enter into clinical trials against cancer. Besides therapeutic potential, the CRISPR-Cas9
tool can also be applied to generate genetically inhibited animal models for drug
discovery and development. This comprehensive review paper discusses the origin of
CRISPR-Cas9 systems and their therapeutic potential against various genetic disorders,
including cancer, allergy, immunological disorders, Duchenne muscular dystrophy, cardiovascular
disorders, neurological disorders, liver-related disorders, cystic fibrosis, blood-related
disorders, eye-related disorders, and viral infection. Finally, we discuss the different
challenges, safety concerns, and strategies that can be applied to overcome the obstacles
during CRISPR-Cas9-mediated therapeutic approaches.
</p>