Virus-based gene therapy research

Virus-based gene therapy uses engineered viral vectors — including adeno-associated viruses (AAV), lentiviruses, and adenoviruses — to deliver functional genetic material into human cells, either correcting faulty genes or introducing new therapeutic ones. The approach has produced genuine clinical successes, particularly in treating inherited immunodeficiencies and blood disorders through hematopoietic stem cell modification, where a patient's own stem cells are corrected ex vivo before reinfusion. Researchers are also developing oncolytic viruses, which are engineered to selectively replicate inside and destroy tumor cells, adding an immunotherapy dimension to the toolkit. Central open questions include how to achieve durable, controlled transgene expression without triggering immune responses, and how to ensure genomic integration occurs safely without disrupting cancer-suppressor genes.

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Keywords

Gene TherapyViral VectorsAAVOncolytic VirusHematopoietic Stem CellImmunodeficiency