Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study

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Journal

The Lancet

Topics

Medicine

Internal Medicine

Ophthalmology

Pediatrics

Molecular Biology

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DOI

10.1016/s0140-6736(24)01447-8