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

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

Published:2014-10 Issue:9953 Volume:384 Page:1513-1520
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Koenekoop Robert K,Sui Ruifang,Sallum Juliana,van den Born L Ingeborgh,Ajlan Radwan,Khan Ayesha,den Hollander Anneke I,Cremers Frans P M,Mendola Janine D,Bittner Ava K,Dagnelie Gislin,Schuchard Ronald A,Saperstein David A

Publisher

Elsevier BV

Subject

General Medicine

Reference31 articles.

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2. An overview of Leber congenital amaurosis: a model to understand human retinal development;Koenekoop;Surv Ophthalmol,2004

3. Leber congenital amaurosis: genes, proteins and disease mechanisms;den Hollander;Prog Retin Eye Res,2008

4. Mutations in the gene encoding lecithin retinol acyltransferase are associated with early-onset severe retinal dystrophy;Thompson;Nat Genet,2001

5. Mutations in RPE65 cause autosomal recessive childhood-onset severe retinal dystrophy;Gu;Nat Genet,1997

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