AAV Gene Therapy for MPS VI Corneal Blindness
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Organization
UNC - Chapel Hill
Award Date
Award Amount
100,000
Program Name
Translational Research Grant
Program Category
Research and Equipment Support
Principal Investigator
Matthew Hirsch
Fiscal Year
2020
Region
Research Triangle
Project Description:
All patients with Mucopolysaccharidosis VI (MPS VI) disease experience progressive vision loss due to corneal clouding. This project evaluate a gene therapy approach towards the development of a single dose therapeutic to prevent or reverse MPS VI vision loss.