Profile

The Thangavel laboratory has the goal of developing gene and cell therapy to treat patients with genetic and infectious diseases. Our primary focus is on gene editing the patient's hematopoietic stem cells for autologous stem cell transplantation to correct the underlying genetic cause. The diseases we target include Sickle cell Anemia, B-thalassemia, Primary immunodeficiency disorder, and HIV. We are also working on developing novel gene-delivery platforms, culture conditions, and target populations to create a cost-effective gene editing therapy.

Current Focus Areas

  • Developing gene edited Hematopoietic stem and progenitor cells for gene therapy for beta-hemoglobin disorders and HIV

Selected Publications

  • Venkatesan, V., Christopher, A. C., Rhiel, M., Kumar, M., Azhagiri, K., Babu, P., Walavalkar, K., Saravanan, B., Andrieux, G., Rangaraj, S., Srinivasan, S., Karuppusamy, K. v, Jacob, A., Bagchi, A., Pai, A. A., Nakamura, Y., Kurita, R., Balasubramanian, P., Pai, R., … Thangavel, S. (2023). Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies. Molecular Therapy - Nucleic Acids, 32, 671–688. https://doi.org/10.1016/J.OMTN.2023.04.024 Karuppusamy, K. v., Demosthenes, J. P., Venkatesan, V., Christopher, A. C., Babu, P., Azhagiri, M. K., Jacob, A., Ramalingam, V. V., Rangaraj, S., Murugesan, M. K., Marepally, S. K., Varghese, G. M., Srivastava, A., Kannangai, R., & Thangavel, S. (2022). The CCR5 Gene Edited CD34+CD90+ Hematopoietic Stem Cell Population Serves as an Optimal Graft Source for HIV Gene Therapy. Frontiers in Immunology, 13, 792684. https://doi.org/10.3389/FIMMU.2022.792684/BIBTEX 6. Christopher, A. C., Venkatesan, V., Karuppusamy

Skills & Proficiency

Gene therapy Hematopoietic stem cells gene editing Beta-hemoglobinopathies HIV Sickle cell anemia B-thalassemia Extracellular vesicles Homology directed repair C