Innovations in Gene Therapy: Revolutionizing Treatment for Genetic Disorders and Diseases

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This 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.

Year : 2026 | Volume : 16 | 01 | Page :
    By

    Disha J. Nagdev,

  • Beauty A. Yadav,

  • Krinal A. Patel,

  • Nidhi K. Parmar,

  • Bisojeet R. Khandai,

  1. Student, Department of Pharmacy Practice, Maliba Pharmacy College, Uka Tarsadia University, Bardoli, Gujarat, India
  2. Student, Department of Pharmacy Practice, Maliba Pharmacy College, Uka Tarsadia University, Bardoli, Gujarat, India
  3. Student, Department of Pharmacy Practice, Maliba Pharmacy College, Uka Tarsadia University, Bardoli, Gujarat, India
  4. Student, Department of Pharmacy Practice, Maliba Pharmacy College, Uka Tarsadia University, Bardoli, Gujarat, India
  5. Student, Department of Pharmacy Practice, Maliba Pharmacy College, Uka Tarsadia University,, Bardoli, Gujarat, India

Abstract

Gene therapy is a revolutionary intervention in the treatment of genetic diseases since it is an intervention that aims at the very source of diseases, through editing and modification of genes. More recent innovations in gene editing systems, such as CRISPR-Cas9, CRISPR-Cas12, and prime editing, have greatly increased the accuracy and effectiveness of gene therapies, enabling more specific therapies. The opportunities of personalized medicine are also extended by the creation of RNA-based
therapies and the use of artificial intelligence (AI) and machine learning, which will allow customizing treatments to the specifics of the genetic makeup. Despite these advances, issues like delivery systems, immune reactions, and regulatory barriers, still remain as barriers to the universal use of gene therapies. Nevertheless, the current clinical trials on diseases, such as Cystic Fibrosis, Duchenne Muscular Dystrophy, and Sickle Cell Anemia, and the possibility to cure such complicated illnesses as cancer and neurodegenerative diseases support the increasing role of gene therapy in contemporary medicine. Gene therapy is the future, which will transform the way diseases are treated around the world.

Keywords: Artificial intelligence, cancer, clinical trials, CRISPR-Cas9, gene editing, gene therapy, genetic disorders, neurodegenerative diseases, personalized medicine, RNA-based therapies

How to cite this article:
Disha J. Nagdev, Beauty A. Yadav, Krinal A. Patel, Nidhi K. Parmar, Bisojeet R. Khandai. Innovations in Gene Therapy: Revolutionizing Treatment for Genetic Disorders and Diseases. Research and Reviews : A Journal of Biotechnology. 2026; 16(01):-.
How to cite this URL:
Disha J. Nagdev, Beauty A. Yadav, Krinal A. Patel, Nidhi K. Parmar, Bisojeet R. Khandai. Innovations in Gene Therapy: Revolutionizing Treatment for Genetic Disorders and Diseases. Research and Reviews : A Journal of Biotechnology. 2026; 16(01):-. Available from: https://journals.stmjournals.com/rrjobt/article=2026/view=239287


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Ahead of Print Subscription Review Article
Volume 16
01
Received 27/01/2026
Accepted 12/02/2026
Published 27/03/2026
Publication Time 59 Days
74

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