Purpose An intravitreally injected antisense oligonucleotide, sepofarsen, was designed to
modulate splicing within retinas of patients with severe vision loss due to deep intronic c …

Leber congenital amaurosis due to CEP290 ciliopathy is being explored by treatment with
the antisense oligonucleotide (AON) sepofarsen. One patient who was part of a larger cohort …

ABSTRACT Introduction Antisense oligonucleotides (ASOs) represent a class of drugs
which can be rationally designed to complement the coding or non-coding regions of target …

Purpose: LCA10 is a severe, degenerative inherited retinal disease resulting in childhood
blindness, which has no treatment. The most common causative mutation is p. Cys998X in …

Purpose: LCA10 is a severe, degenerative inherited retinal disease resulting in childhood
blindness, which has no treatment. Sepofarsen, an RNA antisense oligonucleotide designed …

CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease
resulting in childhood blindness. Sepofarsen is an RNA antisense oligonucleotide targeting …

Leber congenital amaurosis (LCA) is a severe disorder resulting in visual impairment
usually starting in the first year of life. The most frequent genetic cause of LCA is an intronic …

Leber congenital amaurosis type 10 (LCA10) is a severe inherited retinal dystrophy
associated with mutations in CEP290. The deep intronic c. 2991+ 1655A> G mutation in …

Leber congenital amaurosis is a severe hereditary retinal dystrophy responsible for neonatal
blindness. The most common disease-causing mutation (c. 2991+ 1655A> G; 10–15%) …

Leber congenital amaurosis (LCA) is the most severe form of inherited retinal degeneration,
with an onset in the first year of life. The most frequent mutation that causes LCA, present in …