Institute of Child Health

Society for Mucopolysaccharide Diseases
MPS House, Repton Place, White Lion Road, Amersham, Buckinghamshire, HP7 9LP, UK
Tel: 0845 389 9901 Fax: 0845 389 9902
National Registered Charity No. 287034

Involvement of the Blood Brain Barrier in MPS III A and MPS III B
1 year project supported by a grant from the Shauna Gosling Charitable Trust £45,959
The specific aims of this project is the comparison and quantification of the blood-brain barrier (BBB) permeability to solutes of defined molecular weight and physio-chemical characteristics in two mouse models of Sanfilippo Syndrome (MPS III A and MPS III B) and in normal mice. This information will give some guidance, firstly, as to how extensively the BBB may be compromised in human MPS III A and MPS III B and how the transport function may alter. Secondly, it may tell us whether or not there is progression of loss of the integrity of the BBB with advancing age and in this way, help to define optimal ages at which treatment might be initiated. Confirmation that the BBB is compromised or altered in MPS III A and MPS III B mice will fundamentally change thinking on the development and treatment of this particular disorder, and may also have implications for a significant amount of other lysosomal storage disorders.