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 equipping cells with new instructions. The approach has moved from theory to clinical reality in diseases like inherited immunodeficiencies, where hematopoietic stem cell gene therapy has produced lasting corrections in patients who previously had few options. Researchers are also developing oncolytic viruses, which are designed to selectively replicate inside and destroy tumor cells, expanding the strategy beyond inherited disease into cancer treatment. Central open questions involve controlling where viral payloads integrate into the genome, achieving durable transgene expression without triggering immune responses, and scaling manufacturing to meet the precision each patient's treatment demands.

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139,857

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Keywords

Gene TherapyViral VectorsAAVOncolytic VirusHematopoietic Stem CellImmunodeficiency