Volume 3 Supplement 1
Mouse positioning device for blood brain barrier FUS exposure
© Gong et al; licensee BioMed Central Ltd. 2015
Published: 30 June 2015
Gaucher’s disease is an inherited metabolic disorder that results in a deficiency of the enzyme glucocerebrosidase, which acts on the glycolipid glucocerebroside. This causes a rapid accumulation of glucocerebroside in affected persons, which manifests in organ malfunctions and disfiguration, swelling, and severe neurologic complications, among other symptoms. Enzyme replacement therapy can manage the symptoms but when the brain is affected, the treatment is not effective since the enzymes cannot cross the blood brain barrier (BBB). Imaging of enzyme replacement therapy has shown that only insignificant amounts reach the brain. In this work, we intend to evaluate brain uptake of enzyme replacement after BBB opening mediated by focused ultrasound (FUS) to validate this delivery technique for the treatment of neurologic complications caused by Gaucher’s disease. Our goal is to develop a system for Blood Brain Barrier delivery that can be followed up using positron emission tomography (PET) imaging. It will then be used to create and validate the delivery after intravenous injection of radiolabelled enzymes.
Results and conclusions
Our group would like to thank the Focused Ultrasound Foundation and the Thunder Bay Regional Research Institute for providing funding for our research.
This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.