2015

2015 Research Grants:

• General Grant

Targeting mTORCI and autophagy pathways to rescue the skeletal phenotype in MPS mouse models

Dr. Carmine Settembre
Telethon Institute of Genetics and Medicine of Fondazione Telethon
Pozzuoli, Italy

• MPS I

Validation of small molecule therapeutic leads for treatment of MPS I disease

Dr. Allison R. Kermode, Professor
Simon Fraser University
Burnaby, BC Canada

• MPS III

AAV2/8 medicated expression of modified sulphamidase: Liver targeting for improved secretion and brain deliver. Pre-clinical study in the Huntaway dog

Dr. Kim Hemsley, Dr. Alessandro Fraldi, and Professor Robert D. Jolly
Lysosomal Diseases Research Unit
Adelaide, SA, Australia

• MPS IVA

Creating new tools for understanding skeletal disease in MPS IVA

Dr. Ainslie Derrick-Roberts
Genetics and Molecular Pathology
North Adelaide, SA, Australia

• MPS I

Igor Nestrasil, MD
University of Minnesota
Mucopolysaccharidoses pilot for $50,500
Microstructural and functional MRI signatures in patients with MPS I