Official websites use .gov
A
.gov website belongs to an official
government organization in the United States.
Secure .gov websites use HTTPS
A lock (
) or https:// means you've safely
connected to the .gov website. Share sensitive
information only on official, secure websites.
Schematic overview of the underlying concept. Intravenously injected AAV-BR1-CAG-HEXA/B vectors transduce brain endothelial cells. The vector DNA is transcribed, leading to the production of β-hexosaminidase which is released into the brain parenchyma. Although not shown in this study, neurons probably endocytose β-hexosaminidase, which enables the degradation of gangliosides and rescues the cells from neurodegeneration.
