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2016

2016 Research Grants:

ML II

To evaluate AAV Gene Therapy in the feline model of ML II

Dr. Allison Bradbury, University of Pennsylvania
Dr. Charles Vite, University of Pennsylvania
Dr. Steven Gray, University of North Carolina at Chapel Hill

MPS IIIA

AAV2/8-mediated expression of modified sulphamidase, liver targeting for improved secretion and brain delivery in IIIA Huntaway dog
Dr. Kim Hemsley
South Australia Health and Medical Research Institute
Adelaide, South Australia

MPS IVA

Gene Therapy for MPS IVA

Dr. Kazuki Sawamoto, Dr. Shunji Tomatsu
Nemours/Alfred I. duPont Hospital for Children
Wilmington, DE

MPS VII

Therapeutic Targeting of Wnt/β-Catenin Signaling to Improve Bone Formation in MPS VII

Dr. Lachlan J. Smith
University of Pennsylvania
Philadelphia, PA

2014

2014 Research Grants:

The National MPS Society allocated $509,000 in grant funding for 2014 which includes the second year funding for grants awarded in 2013, the unspent MPS III funds from 2012 and the 2014 grants. The funding we provide is critical as we move forward with our mission to find cures for MPS and ML. We received 20 letters of intent from researchers around the world for the General, MPS II, MPS IVA and MPS VI grants. After reviewing those letters, our Scientific Advisory Board review committee requested full grant proposals from eight researchers.

The board of directors allocated $100,000 to Abeona Therapeutics which has licensed the MPS IIIA and MPS IIIB gene therapy technology from Nationwide Children’s Hospital. Funds already raised by Abeona have been funneled to Nationwide for MPS III drug manufacturing and preclinical research plus two INDs (investigational new drug applications). The financial distribution from the Society will help move the clinical trial forward.

We also provided $25,000 to support the Lysosomal Disease Network’s NIH grant research goals. The funding is designed for the Neuroimaging Core, which will benefit the four MPS projects. An additional $8,000 was offered for an ML grant in partnership with ISMRD (International Society for Mannosidosis and Related Diseases). A $10,000 partnership grant with the Ryan Foundation funds the University of MN project “Longitudinal Studies of Brain Structure and Function in MPS Disorders.” We also provide funding for post-doctoral fellows to attend the American Society and Gene and Cell Therapy conference.

General Grant

The role of angiotensin II-mediated inflammation in MPS I vascular disease: a study of pathophysiologic mechanism and evaluation of angiotensin receptor blockade therapy

Moin Vera, MD, PhD
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Torrance, CA

MPS II

AAV mediated gene transfer to the CNS for MPS II

Scott McIvor, PhD
University of Minnesota
Minneapolis, MN

MPS III

Overcoming limitations inherent in sulfamidase to improve MPS IIIA gene therapy

Beverly Davidson, PhD
The Children’s Hospital of Philadelphia

MPS VI

Pentosan Polysulfate and GAGs in MPS

Icahn School of Medicine at Mount Sinai
New York, NY

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Meet Our Staff

Society Funded Research Archive

2016 Research Grants:

2014 Research Grants:

Our Mission