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Becker's Healthcare September 10, 2024
In a phase 1/2 trial, researchers tested ATSN-101, a gene therapy for Leber congenital amaurosis 1, a rare genetic disorder that typically causes blindness in early childhood.
Fifteen patients with mutations in the GUCY2D gene were treated with increasing doses of ATSN-101. Overall, the subretinal therapy was well tolerated, with...
