A viral vector delivery system that permits real-time, direct MRI monitoring of vector distribution within the brain was presented at the 10th Annual Meeting of the American Society of Gene Therapy (ASGT) in Seattle.
Success of gene therapy for neurological disorders depends on precise and reproducible intracranial delivery of viral vectors that encode therapeutic genes. However, optimal viral vector delivery into the brain is challenging and brain distribution of viral vectors is uncertain. Parameters required for optimal delivery such as volume, rate of infusion, and cannula placement had to be defined experimentally to predict desired coverage of each anatomical target to avoid introducing the vector to regions outside the target, including cerebrospinal fluid (CSF).
A team of scientists, led by Dr. Krystof Bankiewicz, University of California-San Francisco, presented this significant advance, which allows, for the first time, to adjust parameters of vector infusion while delivering gene therapy, giving surgeon full control over gene transfer technology.
To demonstrate applicability of this technology, adeno-associated viral (AAV) vectors were co-administered together with gadolinium (MRI-contrast agent) nano-liposomes into several brain regions in monkeys under MRI control.
With this technology, AAV delivery can be monitored in real time during AAV administration, thus, precise gene expression over small and large targets can be assured increasing safety and efficacy of AAV administration to the brain and spinal cord.
The American Society of Gene Therapy is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of gene and related cell and nucleic acid therapies and the promotion of professional and public education in the field. For more information, visit