AAV Gene Therapy for MPS1-associated Corneal BlindnessMelisa Vance(University of North Carolina at Chapel Hill), Matthew L. Hirsch(University of North Carolina at Chapel Hill)Scientific ReportsFebruary 22, 201610.1038/srep22131Cited by 50SaveCiteExport RISWatch citationsRelated PapersA chimeric anti-vascularization immunomodulator prevents high-risk corneal transplantation rejection via ex vivo gene therapy|Molecular Therapy|2024|9AAV vector transduction restriction and attenuated toxicity in hESCs via a rationally designed inverted terminal repeat|Nucleic Acids Research|2025|7