CRISPR-CAS9 APPLICATIONS IN FUNCTIONAL GENOMICS AND GENE THERAPY
DOI:
https://doi.org/10.71465/hjmri.254Keywords:
CRISPR-Cas9, Genome Editing, Functional Genomics, Gene TherapyAbstract
Targeted manipulation of DNA with unprecedented precision, efficiency, and versatility. Its impact spans functional genomics—enabling gene knockout, activation, and repression screens—to transformative applications in gene therapy, including the correction of genetic mutations in inherited disorders. This paper explores the molecular mechanism of CRISPR-Cas9, its applications in functional genomic analysis, its therapeutic relevance, challenges in delivery systems, and ethical implications. It also highlights the contributions of Pakistani research institutions in this growing domain and outlines future directions for clinical translation.
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