International Journal of Research in Pharmacy and Allied Science

ISSN: 2583-6544   |  Issues per year: Monthly  |   Indexed In: Google Scholar, CORE, ScienceOpen, Researchgate

ISSN:2583-6544

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CRISPR-Cas Systems in Targeted Drug Delivery and Gene Therapy: An Emerging Approach to Precision Medicine

Author(s): Mohd Uzair1, Shabnam Ain*2, Qurratul Ain3, Babita Kumar4, Vipin Kumar5, Sneha Pandey6, Ajeet and Cutee7

Email(s): 1shabnam.ain@sanskar.org

Address:

    Sanskar College of Pharmacy and Research, Ghaziabad, Uttar Pradesh.

Published In:   Volume - 5,      Issue - 2,     Year - 2026

DOI: https://doi.org/10.71431/IJRPAS.2026.5206  

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ABSTRACT:
The CRISPR-Cas system has revolutionized genetic engineering by offering precise genome editing with unparalleled accuracy. Its integration into targeted drug delivery and gene therapy represents a transformative approach for treating genetic disorders, cancers, and infectious diseases. Originally identified as part of a bacterial immune defense mechanism, CRISPR-Cas has evolved into a versatile tool for site-specific genome editing, enabling correction of disease-causing mutations with minimal off-target effects. In drug delivery, CRISPR-based platforms are being engineered to direct therapeutic agents specifically to diseased tissues or cells, thereby reducing systemic toxicity while enhancing efficacy. Advanced carriers such as nanoparticles, liposomes, and viral and non-viral vectors are under exploration to improve the efficiency and precision of CRISPR component delivery. In the realm of gene therapy, CRISPR technologies show significant promise for treating conditions such as sickle cell anemia, cystic fibrosis, muscular dystrophy, and various malignancies by rewriting defective genes or introducing protective modifications within the genome. Despite this promise, challenges such as delivery limitations, immunogenicity, and ethical concerns, particularly regarding germline editing, remain critical. Regulatory frameworks are evolving to ensure safe clinical translation. Overall, crispr-cas technologies are expected to play a pivotal role in advancing personalized medicine, offering hope for conditions once considered untreatable. This review highlights recent breakthroughs, evaluates delivery strategies, and discusses future directions toward safe and effective clinical application

Keywords:

Cite this article:
Mohd Uzair, Shabnam Ain, Qurratul Ain, Babita Kumar, Vipin Kumar, Sneha Pandey, Ajeet and Cutee. CRISPR-Cas Systems in Targeted Drug Delivery and Gene Therapy: An Emerging Approach to Precision Medicine. IJRPAS, February 2026; 5(2): 66-87.DOI: https://doi.org/https://doi.org/10.71431/IJRPAS.2026.5206


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