Author(s):
Preksha Patel, Anuradha Prajapati, Sachin B. Narkhede, Shailesh Luhar, Pooja Patel
Email(s):
prekshapatel2504@gmail.com , anuradha@ssgsalvav.in , sachinnarkhedein@gmail.com , sv_pharma2008@yahoo.com , poojapatelwap@gmail.com
DOI:
10.52711/0975-4377.2025.00038 
Address:
Preksha Patel, Anuradha Prajapati, Sachin B. Narkhede, Shailesh Luhar, Pooja Patel
Smt BNB Swaminarayan Pharmacy College, Salvav, Vapi – 396191.
*Corresponding Author
Published In:
Volume - 17,
Issue - 4,
Year - 2025
ABSTRACT:
The Aim of this article is to review CRISPR-based drug delivery systems and their therapeutic applications in gene therapy, focusing on advancements, challenges, and future prospects. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology has emerged as a transformative tool in gene therapy, enabling precise and efficient genome editing with unprecedented ease compared to earlier platforms such as zinc finger nucleases and TALENs. By leveraging the Cas nuclease system, CRISPR enables targeted modification of disease-causing genes, offering promising therapeutic interventions for genetic disorders, cancers, and infectious diseases. However, the clinical translation of CRISPR-based therapeutics critically depends on the development of safe, efficient, and tissue-specific delivery systems. Current strategies encompass viral vectors (e.g., adeno-associated viruses, lentiviruses) and non-viral carriers (e.g., lipid nanoparticles, polymeric systems, cell-penetrating peptides), each with unique advantages and limitations in terms of delivery efficiency, cargo capacity, immunogenicity, and off-target effects. Advances in nanotechnology and biomaterials have further enabled targeted and stimuli-responsive delivery, enhancing therapeutic precision while minimizing systemic toxicity. Furthermore, CRISPR-based modalities such as base editing, prime editing, and CRISPR interference/activation expand the therapeutic landscape beyond simple gene knockout or repair. Despite remarkable progress, challenges remain in optimizing delivery to specific tissues, controlling off-target activity, and addressing ethical and regulatory concerns. This review provides a comprehensive overview of CRISPR-based drug delivery systems, recent preclinical and clinical advancements, and future perspectives in gene therapy, emphasizing innovations that bridge the gap between experimental success and clinical application.
Cite this article:
Preksha Patel, Anuradha Prajapati, Sachin B. Narkhede, Shailesh Luhar, Pooja Patel. Crispr-Based Drug Delivery and Therapeutics in Gene Therapy: A Comprehensive Review. Research Journal of Pharmaceutical Dosage Forms and Technology. 2025; 17(4):273-8. doi: 10.52711/0975-4377.2025.00038
Cite(Electronic):
Preksha Patel, Anuradha Prajapati, Sachin B. Narkhede, Shailesh Luhar, Pooja Patel. Crispr-Based Drug Delivery and Therapeutics in Gene Therapy: A Comprehensive Review. Research Journal of Pharmaceutical Dosage Forms and Technology. 2025; 17(4):273-8. doi: 10.52711/0975-4377.2025.00038 Available on: https://rjpdft.com/AbstractView.aspx?PID=2025-17-4-8
10. REFERENCES:
1. Mukund M. Pache, Rutuja R. Pangavhane, Siddhi V. Nikam, Ramdas B. Rode, Avinash B. Darekar. CRISPR-Cas9 in Pharmaceutical Research: Applications, Challenges, Ethical Considerations and Future Directions. Asian Journal of Pharmacy and Technology. 2025; 15(3): 296-4.
2. Ali Adel Dawood, Bassam Ismaeel Jasim. The CRISPR Genome Editing Process is an Effective Advancement of Short-Term Cancer Treatment. Res. J. Pharma. Dosage Forms and Tech. 2021; 13(1): 54-56.
3. PM. Patil, PD Chaudhari, Megha Sahu, NJ Duragkar. Review Article on Gene Therapy. Research J. Pharmacology and Pharmacodynamics. 2012; 4(2): 77-83.
4. Sulochana Dutta, B. Ray, Shibani Raut, C. K. Sahoo. Nonviral Gene Therapy: Technology and Application. Research J. Science and Tech. 2021; 13(1): 13-22.
5. Ahir Hemini Mukeshbhai, Anuradha P. Prajapati, Sachin B. Narkhede, Shailesh Luhar. Gene Therapy for Regenerative Medicine: Advances and Challenges. Research Journal of Science and Technology. 2025; 17(1): 31-4.
6. Shivanika Mani, Brindha Devi P. Study on Genetically Engineered Vesicular Stomatitis Virus for the Application of Treating Malignant Diseases using Gene Therapy. Research J. Pharm. and Tech. 2019; 12(11): 5371-5378.
7. R. B. Shinde, A. H. Hosmani, M. A. Shende, R. J. Jarag, Y. S. Thorat. Preparation, statistical optimization, in-vitro evaluation and characterization of solid lipid nanoparticles of an anti-retroviral drug Nevirapine. Research Journal of Pharmacy and Technology. 2023; 16(8): 3899-8.
8. Amica Panja, Ashish Kumar Mishra, Mahesh Dash, Narendra Kumar Pandey, Sachin Kumar Singh, Bimlesh Kumar. Solid Lipid Nanoparticles: A Promising Novel Carrier. Research Journal of Pharmacy and Technology 2022; 15(12): 5879-5.
9. Anshul Sharma, Keshav Dhiman, Anshul Sharma, Kamya Goyal, Vinay Pandit, M. S. Ashawat, Shammy Jindal. Application of Nanocarrier in Drug development with special Emphasis on Liposomes: A Review. Asian Journal of Pharmacy and Technology. 2022; 12(4): 320-8.
10. Ganesh G. Dhakad, Sangita P. Shirsat, Kaveri P. Tmabe, Neha R. Jaiswal. Review on Gene Therapy on Cancer. Research Journal of Pharmacology and Pharmacodynamics. 2022; 14(1):37-2.
11. Novel Carrier. Research Journal of Pharmacy and Technology. 2022; 15(12): 5879-5.
12. Anshul Sharma, Keshav Dhiman, Anshul Sharma, Kamya Goyal, Vinay Pandit, M. S. Ashawat, Shammy Jindal. Application of Nanocarrier in Drug development with special Emphasis on Liposomes: A Review. Asian Journal of Pharmacy and Technology. 2022; 12(4): 320-8.
13. Ganesh G. Dhakad, Sangita P. Shirsat, Kaveri P. Tmabe, Neha R. Jaiswal. Review on Gene Therapy on Cancer. Research Journal of Pharmacology and Pharmacodynamics. 2022; 14(1): 37-2.