Gene therapy for GUCY2D restores vision in first-ever trial for rare, inherited blindness known as Leber congenital amaurosis type I, or LCA1
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#GeneTherapy #GUCY2D #VisionRestoration #LCA1 #RareBlindness
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#GeneTherapy #GUCY2D #VisionRestoration #LCA1 #RareBlindness
The Lancet
Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2…
ATSN-101 is well tolerated 12 months after treatment, with no drug-related serious
adverse events. Clinically significant improvements in retinal sensitivity were sustained
in patients receiving the high dose.
adverse events. Clinically significant improvements in retinal sensitivity were sustained
in patients receiving the high dose.
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