Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect

Cideciyan, AV; Jacobson, SG; Drack, AV; Ho, AC; Charng, J; Garafalo, AV; Roman, AJ; ... Russell, SR; + view all Cideciyan, AV; Jacobson, SG; Drack, AV; Ho, AC; Charng, J; Garafalo, AV; Roman, AJ; Sumaroka, A; Han, IC; Hochstedler, MD; Pfeifer, WL; Sohn, EH; Taiel, M; Schwartz, MR; Biasutto, P; Wit, WD; Cheetham, ME; Adamson, P; Rodman, DM; Platenburg, G; Tome, MD; Balikova, I; Nerinckx, F; Zaeytijd, JD; Van Cauwenbergh, C; Leroy, BP; Russell, SR; - view fewer (2018) Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect. Nature Medicine 10.1038/s41591-018-0295-0 . (In press).

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