Facilitating and Enhancing CNS Entry of Systemically Delivered AAV Vectors for MPS IIIA, IIIB in Mice

September 30, 2019

Uniform gene delivery to the central nervous system (CNS) remains one of the most intractable challenges to the development of effective gene therapies for a wide range of neurological diseases, including MPS III (Sanfilippo Syndrome).

While the brain microvasculature offers the greatest accessibility to the cells throughout the CNS, the blood-brain barrier (BBB) presents the paramount obstacles to systemically-delivered gene therapy products.

A few viral vectors – notably AAV serotype 9 and closely-related serotypes – can cross the BBB, but only in quantities limited by receptor-mediated endothelial transcytosis.

Cure Sanfilippo Foundation is providing two-year grant support to the team of Dr. Haiyan Fu at the University of North Carolina at Chapel Hill to develop a more effective systemic rAAV9 gene delivery approach for MPS IIIA and IIIB.

This project aims to lead to the development of safe and effective approaches that can enhance the neurological benefit of systemic AAV9 gene delivery for MPS IIIA and IIIB.

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