Scholarly Article

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

Imran Khan Yousafzai, Aqsa Mehreen, Nadia Noreen, Nayab Ahsan, Shanzay Malik, Anza Mnahal, Sanam Rehman, Hawaida Ahmad, Khadija Tariq

2025-12-17 · Advances in Modern Biomedicine · Cultech Publishing Sdn. Bhd.

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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.

Keywords

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

Citation Details

Advances in Modern Biomedicine, pp. 11-22