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
Home | Diseases | Advocacy Support | Information Resource | Childhood Wood | Events | Latest News
Get Involved! | Jeans for Genes | Register an MPS Sufferer | Become a Friend of MPS
Contact Us | International Contacts | Links | Research
University of Gdansk, Royal Manchester Children's Hospital
The Development of Gene Expression-Targetted Isoflavone Therapy (GET IT) for Mucopolysaccharidoses
3 year grant £160,000
The aim of this project is development of principles of a new therapy for MPS III, Sanfilippo Disease, based on reduction of the substate (heparan sulphate) that cannot be degraded in lysosomes of affected patients due to a defect in one of the enzymes. Although enzyme replacement therapy (ERT) has recently been available for MPS I and MPS VI and one may assume that analogous therapy may be available for some other MPS tyles relatively soon, it is little chance that this kind of treatment may be effective for MPS III. This is due to the severe neurological problems in MPS III and inefficient delivery of proteins including those used in ERT, to central nervous system, because of the blood-brain barrier. Therefore the aim is to develop an alternative therapy for MPS III.
.
