Gene Therapy Approaches in Hematological Malignancies: Current Strategies and Future Directions

Authors

  • Imran Khan Yousafzai Department of Biological Sciences, National University of Medical Science, Rawalpindi, Punjab, Pakistan
  • Aqsa Mehreen Department of Biological Sciences, National University of Medical Science, Rawalpindi, Punjab, Pakistan
  • Nadia Noreen Department of Biological Sciences, Abasyn University, Islamabad, Pakistan
  • Nayab Ahsan Department of Biological Sciences, National University of Medical Science, Rawalpindi, Punjab, Pakistan
  • Shanzay Malik Department of Biological Sciences, National University of Medical Science, Rawalpindi, Punjab, Pakistan
  • Anza Mnahal Department of Biological Sciences, National University of Medical Science, Rawalpindi, Punjab, Pakistan
  • Sanam Rehman Department of Biomedical Engineering and Sciences, School of Mechanical and Manufacturing Engineering, National University of Sciences & Technology, Islamabad, Pakistan
  • Hawaida Ahmad Department of Biological Sciences, National University of Medical Science, Rawalpindi, Punjab, Pakistan
  • Khadija Tariq Department of Biological Sciences, National University of Medical Science, Rawalpindi, Punjab, Pakistan

DOI:

https://doi.org/10.63623/c718ab40

Keywords:

Hematological malignancies, Gene therapy, CRISPR-Cas9, CAR-T therapies, Gene delivery

Abstract

Hematological malignancies, such as leukemia, lymphoma, and myeloma, are cancers originating in the bone marrow and lymphatic system, marked by the uncontrolled growth of blood cells. Gene therapy offers a revolutionary treatment approach by directly targeting genetic mutations and boosting the body’s anti-tumor immune responses. This study aims to provide a comprehensive overview of hematological cancers and explores the current and emerging gene therapy strategies that are being investigated for their treatment. It also provides a detailed overview of gene delivery systems, their principles, types, and recent advancements in gene therapy as a potential treatment strategy. It provides a detailed analysis of gene delivery systems, therapeutic modalities, and clinical applications, with a focus on CAR-T, CRISPR-Cas9, and RNA interference (RNAi) technologies. It highlights recent advances in gene delivery, including viral and non-viral vector systems, along with advanced techniques like gene editing and gene silencing. Special attention is given to cutting-edge techniques such as CRISPR-Cas9, CAR-T therapies, and RNA interference. The article concludes by discussing current hurdles and prospects of gene therapy, emphasizing its immense potential to transform the treatment for hematological cancer.

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Published

2025-11-26

Issue

Vol. 1 No. 3 (2025)

Section

Articles

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Copyright (c) 2025 Imran Khan Yousafzai, Aqsa Mehreen, Nadia Noreen, Nayab Ahsan, Shanzay Malik, Anza Mnahal, Sanam Rehman, Hawaida Ahmad, Khadija Tariq (Author)

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