2016

2016 Research Grants:

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

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

Gene Therapy for MPS IVA

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

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

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

Our Mission

The National MPS Society exists to cure, support and advocate for MPS and ML.

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