Complexity and diversity of bioanalytical support for gene therapy modalities
Gorovits B | Bioanalysis, 13(2), 65 – 68, (2021)
Keywords: • AAV • adeno-associated virus • bioanalysis • gene therapy • GTx
Ex vivo and in vivo gene therapy (GTx)-based methodologies for treatment of various human conditions are experiencing a clear and dynamic renaissance, with several therapeutic modalities and platforms evaluated preclinically or already in patients. A significant number of gene therapies are developed to treat various cancers or rare genetic diseases. These revolutionary and potentially disease-modifying approaches to medical intervention are intended to work through recruitment of elements of the immune system to attack cancer cells or to correct the root of the underlying condition caused by a single gene defect with a single or limited number of treatments.
