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Researchers have used AAV-based vectors in pre-clinical research and in clinical trials in which AAV-based vectors have demonstrated a good safety profile. AAVs have also demonstrated lasting therapeutic gene expression following a single treatment in preclinical and clinical studies.
Scientists today have used the AAV9 variant, or serotype, of vector in potential product candidates in hemophilia B and Huntington's disease. Research suggests that a greater portion of patients can be effectively treated with AAV9 gene therapy, compared to other AAV-based gene therapies.
