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Showing posts from July, 2023

AAV Gene Therapy: Vector Shedding In Clinical Studies

Vector shedding refers to the release or shedding of viral vectors from cells transduced with the vectors. AAV (adeno-associated virus) is a commonly used viral vector in gene therapy. Studying its fate after the administration to the clinical trial subjects is critical to understanding the shedding route and the duration. Although AAVs are noninfectious, replication incompetent and do not propagate outside the cells, understanding the route and duration of the shedding would enable the mitigation of any potential risk of viral vectors including: spread of AAV vectors beyond treated subjects/patients (both horizontal and vertical transfer) deleterious effects on persons other than the study subjects. Method for Evaluation of Vector Shedding and Clearance (Duration) Vector DNA biodistribution is assessed by qPCR in blood and vector DNA shedding was assessed by qPCR in semen, saliva, urine, and stool. The biodistribution and shedding of potentially infectious vector DNA may be further ch