Jangale Mayuri Navnath,
S. D. Mankar,
- Student, Pravara Rural College of Pharmacy, Pravaranagar, Loni Bk, Maharashtra, India
- Assistant Professor, Pravara Rural College of Pharmacy, Pravaranagar, Loni Bk.,, Maharashtra, India
Abstract
One of the most talked-about topics of the 21st century is gene therapy, which holds the promise of treating many diseases. Current gene therapy research explores a wide range of potential treatments, such as enhancing the body’s immune response to tumors, promoting the formation of new blood vessels in the heart to mitigate heart attacks, and preventing HIV replication in AIDS patients. Gene therapy involves the introduction, alteration, or replacement of genetic material within a patient’s cells to treat or prevent disease. This innovative approach has evolved significantly over the years, overcoming early challenges related to gene delivery, immune responses, and ethical considerations. Advances in viral and non-viral vector systems have improved the efficiency and safety of gene transfer, making it possible to target specific diseases more effectively. Materials and Methods: Relevant articles were thoroughly analyzed and reviewed to assess the historical, current, and future perspectives of gene therapy. Various clinical trials and experimental studies have provided valuable insights into gene-editing techniques, including CRISPR-Cas9, zinc finger nucleases, and RNA interference, all of which have expanded the therapeutic possibilities of gene-based treatments. Unlike traditional gene replacement therapy, craniofacial regeneration aims to utilize genetic vectors as supplementary elements for tissue growth and repair. The combination of viral gene therapy with craniofacial tissue engineering presents a powerful synergy that can greatly improve our capacity to repair and regenerate tissues directly within the body.
Keywords: Gene therapy, genetic disease, perspective, methods of gene therapy, viral and non-viral vectors
[This article belongs to Emerging Trends in Personalized Medicines ]
Jangale Mayuri Navnath, S. D. Mankar. A Review on Design of Gene Therapy and Its Potential for Craniofacial Regeneration. Emerging Trends in Personalized Medicines. 2025; 02(02):-.
Jangale Mayuri Navnath, S. D. Mankar. A Review on Design of Gene Therapy and Its Potential for Craniofacial Regeneration. Emerging Trends in Personalized Medicines. 2025; 02(02):-. Available from: https://journals.stmjournals.com/etpm/article=2025/view=222358
References
- Avery AT, MacLeod CM, McCarty M. Studies on the chemical nature of the substance inducing transformation of pneumococcal types. Induction of transformation by a deoxyribonucleic acid fraction isolated from Pneumococcus type III. J Exp Med. 1944;79:137–58.
- Breaker R. Engineering ligand-responsive gene-control elements: Lessons learned from natural riboswitches. Gene Ther. 2009;16:1189–201.
- Coleman JE, Huentelman MJ, Kasparov S, Metcalfe BL, Paton JF, Katovich MJ, et al. Efficient large-scale production and concentration of HIV-1-based lentiviral vectors for use in vivo. Comp Neurol. 2003;355:479–89.
- Kaufmann KB, Bing H, Galy A, Schambach A, Grez M. Gene therapy on the move. EMBO Mol Med. 2013;5(11):1642–61.
- Wang D, Gao G, Ehmann WC, Rabkin CS, Eyster ME, Goedert JJ. Thrombocytopenia in HIV-infected and uninfected hemophiliacs. Multicenter Hemophilia Cohort Study. Am J Hematol. 1994;54(4):396–300.
- Epstein A. Progress and prospects: biological properties and technological advances of herpes simplex virus type 1-based amplicon vectors. Gene Ther. 2009;16:709–15.
- Evans C, Ghivizzani S, Robbins P. Progress and prospects: genetic treatments for disorders of bones and joints. Gene Ther. 2009;16:944–52.
- Gill D, Pringle A, Hyde S. Progress and prospects: the design and production of plasmid vectors. Gene Ther. 2009;16:165–71.
- Gerick NM, Hagberg M. Definition of historical models of gene function and their relation to students’ understanding of genetics. Sci Educ. 2006;16(7–8):849–81.
- Kaufmann KB, Bing H, Galy A, Schambach A, Grez M. Gene therapy on the move. EMBO Mol Med. 2013;5(11):1642–61.
- Moss JA. Gene therapy review. Radiol Technol. 2014;86(2):155–80; quiz 181–4.
- Mali S. Delivery systems for gene therapy. Indian J Hum Genet. 2013;19(1):3–8.
- Gardlík R, Pálffy R, Hodosy J, Lukács J, Turna J, Celec P. Vectors and delivery systems in gene therapy. Med Sci Monit. 2005;11:RA110–21.
- Robbins PD, Ghivizzani SC. Viral vectors for gene therapy. Pharmacol Ther. 1998;80(1):35–47.
- Nayerossadat N, Maedeh T, Ali PA. Viral and nonviral delivery systems for gene delivery. Adv Biomed Res. 2012;1:27.
- Nichols EK. Human gene therapy. Cambridge (MA): Harvard University Press; 1988.
- McCain J. The future of gene therapy. Biotechnol Healthc. 2005;2(3):52–60.

Emerging Trends in Personalized Medicines
| Volume | 02 |
| Issue | 02 |
| Received | 28/10/2024 |
| Accepted | 22/12/2024 |
| Published | 20/02/2025 |
| Publication Time | 115 Days |
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