An Overview of Gene Therapy

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nThis is an unedited manuscript accepted for publication and provided as an Article in Press for early access at the author’s request. The article will undergo copyediting, typesetting, and galley proof review before final publication. Please be aware that errors may be identified during production that could affect the content. All legal disclaimers of the journal apply.n

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Year : 2025 [if 2224 equals=””]05/10/2025 at 9:33 PM[/if 2224] | [if 1553 equals=””] Volume : 16 [else] Volume : 16[/if 1553] | [if 424 equals=”Regular Issue”]Issue : [/if 424][if 424 equals=”Special Issue”]Special Issue[/if 424] [if 424 equals=”Conference”][/if 424] 03 | Page :

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    Santosh Kumar Saw, Kamalesh Mistry, Dhananjay Mistry, Md. Aftab Alam,

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

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nGene 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.nn

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

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nSantosh Kumar Saw, Kamalesh Mistry, Dhananjay Mistry, Md. Aftab Alam. [if 2584 equals=”][226 wpautop=0 striphtml=1][else]An Overview of Gene Therapy[/if 2584]. Research and Reviews: A Journal of Pharmaceutical Science. 27/07/2025; 16(03):-.

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nSantosh Kumar Saw, Kamalesh Mistry, Dhananjay Mistry, Md. Aftab Alam. [if 2584 equals=”][226 striphtml=1][else]An Overview of Gene Therapy[/if 2584]. Research and Reviews: A Journal of Pharmaceutical Science. 27/07/2025; 16(03):-. Available from: https://journals.stmjournals.com/rrjops/article=27/07/2025/view=0

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Volume 16
[if 424 equals=”Regular Issue”]Issue[/if 424][if 424 equals=”Special Issue”]Special Issue[/if 424] [if 424 equals=”Conference”][/if 424] 03
Received 29/03/2025
Accepted 19/04/2025
Published 27/07/2025
Retracted
Publication Time 120 Days

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