Year : 2025 | Volume : 16 | Issue : 03 | Page : 89 105

Santosh Kumar Saw,

Kamalesh Mistry,

Dhananjay Mistry,

Md. Aftab Alam,

Research Scholar, Department of Pharmacy, Faculty of Pharmaceutical Science, Mewar University, Gangrar, Chittorgarh, Rajasthan, India
Assistant Professor, Department of Pharmacy, Faculty of Pharmaceutical Science, Mewar University, Gangrar, Chittorgarh, Rajasthan, India
Lecturer, Department of Pharmacy, Faculty of Pharmaceutical Science, Mewar University, Gangrar, Chittorgarh, Rajasthan, India
Lecturer, Department of Pharmacy, Faculty of Pharmaceutical Science, Mewar University, Gangrar, Chittorgarh, Rajasthan, India

Abstract

Gene therapy is a revolutionary technique in medical science that seeks to cure or stop illnesses by means of introducing, altering, or quieting genes inside a patient’s cells. Advancements in molecular biology, genetics, and biotechnology have propelled this field forward markedly changed over the last few decades. The underlying idea of gene therapy is to introduce genetic material into target cells via viral or non-viral vectors to rectify faulty genes, increase therapeutic gene expression, or dampen deleterious mutations.Initially researched as a possible treatment for monogenic diseases like hemophilia and cystic fibrosis, gene therapy has grown to tackle more challenging ailments such as several different kinds of cancer, degenerative neurological disorders, and hereditary blindness. Further advances in medical uses have come from technologies including CRISPR-Cas9 as well as in vivo and ex vivo gene transfer. Notwithstanding favorable clinical results, important factors to consider include ethical issues as well as vector-associated toxicity and immune reactions. Governmental agencies such In guaranteeing the safety and effectiveness of gene therapy treatments, the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) have an important part to play. With gene therapy ready to revolutionize contemporary health by providing customized and perhaps curative therapies for many inherited and acquired conditions in light of ongoing scientific development.

Keywords: Gene therapy, CRISPR-Cas9, Monogenic diseases, Vectors, Ethical issues.

[This article belongs to Research and Reviews: A Journal of Pharmaceutical Science ]

How to cite this article:
Santosh Kumar Saw, Kamalesh Mistry, Dhananjay Mistry, Md. Aftab Alam. An Overview of Gene Therapy. Research and Reviews: A Journal of Pharmaceutical Science. 2025; 16(03):89-105.

How to cite this URL:
Santosh Kumar Saw, Kamalesh Mistry, Dhananjay Mistry, Md. Aftab Alam. An Overview of Gene Therapy. Research and Reviews: A Journal of Pharmaceutical Science. 2025; 16(03):89-105. Available from: https://journals.stmjournals.com/rrjops/article=2025/view=228678

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Regular Issue Subscription Review Article

Research and Reviews: A Journal of Pharmaceutical Science

ISSN: 2229-7006

Volume	16
Issue	03
Received	29/03/2025
Accepted	19/04/2025
Published	27/07/2025
Publication Time	120 Days

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