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AAV Gene Therapy for MPS1 Corneal Clouding and Joint Stiffness

Melisa A. Vance, PhD, Matthew Hirsch, PhD, Richard Jude Samulski, PhD, Joanne Kurtzberg, MD, Laurie Goodrich, DVM, PhD ACVS Diplomate, Llanga Telmo, Bsc
Journal: Blood
Blood (2015) 126 (23): 2041.
https://doi.org/10.1182/blood.V126.23.2041.2041
Published: 2015