Results 121 to 130 of about 683 (138)

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

Nature Medicine, 2022
Stephen R Russell, Artur V Cideciyan, Samuel G Jacobson   +2 more
exaly  

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

New England Journal of Medicine, 2008
Francesca Simonelli, Eric A Pierce, Sandro Banfi   +2 more
exaly  

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Lancet, The, 2009
Alberto Auricchio, Eric A Pierce, Francesco Testa   +2 more
exaly  

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

New England Journal of Medicine, 2015
James W B Bainbridge, Anastasios Georgiadis, Freya M Mowat   +2 more
exaly  

Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial

Lancet, The, 2014
Ruifang Sui, Juliana M F Sallum, Radwan Ajlan   +2 more
exaly  

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

New England Journal of Medicine, 2008
James W B Bainbridge, Robert Henderson, Andrew Stockman   +2 more
exaly  

Leber congenital amaurosis: Genes, proteins and disease mechanisms

Progress in Retinal and Eye Research, 2008
Anneke I Den Hollander, Ronald Roepman, Frans P M Cremers   +2 more
exaly  

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

Nature Medicine, 2018
Artur V Cideciyan, Samuel G Jacobson, Allen C Ho   +2 more
exaly  

Review and update on the molecular basis of Leber congenital amaurosis

World Journal of Clinical Cases, 2015
Juan Carlos Zenteno
exaly  

Mutations in NMNAT1 cause Leber congenital amaurosis with early-onset severe macular and optic atrophy

Nature Genetics, 2012
Isabelle Perrault, Sylvain Hanein, Eduardo D Silva   +2 more
exaly