Virus-based gene therapy research

Virus-based gene therapy uses engineered viral particles—most commonly adeno-associated viruses (AAV), lentiviruses, and adenoviruses—to deliver functional genetic material into human cells, either correcting faulty genes or equipping cells with new capabilities. The approach has moved from concept to clinic most visibly in treatments for inherited immunodeficiencies and blood disorders, where modifying hematopoietic stem cells can provide lasting correction after a single intervention. Researchers are actively working to improve how reliably therapeutic genes are expressed over time, how safely viral DNA integrates into the genome without disrupting neighboring genes, and how the immune system's response to viral vectors can be managed or reduced. A parallel direction involves oncolytic viruses—strains engineered to selectively replicate inside and destroy tumor cells—which expands the therapeutic scope well beyond monogenic disease and into oncology.

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141,213

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Keywords

Gene TherapyViral VectorsAAVOncolytic VirusHematopoietic Stem CellImmunodeficiency