Research and Practice in Thrombosis and Haemostasis (Spring 2019) Vol. 3, No. 2, P. 261 Stanford, S.; Pink, R.; Creagh, D.; et al.
A recent study found that screening for preexisting immunity could be important in identifying patients who are most likely to benefit from gene therapy. The study of an adult hemophilia A cohort in the United Kingdom involved testing citrated plasma samples from 100 patients for preexisting activities against adeno-associated virus type 5 (AAV5) and AAV type 8 (AAV8) using AAV transduction inhibition and total antibodies assays. According to the data, 21% of patients had anti-AAV5 antibodies, while 23% had anti-AAV8 antibodies. Additionally, 25% of patients had AAV5 inhibitors and 38% had AAV8 inhibitors. In this cohort, the overall seroprevalence with either assay was 30% against AAV5 and 40% against AAV8. A total of 24% of patients were seropositive for both AAV types. Clinical research may be useful to assess the effects of preexisting immunity on the safety and efficacy of AAV-mediated gene therapy, the researchers report.