Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4-Reference-Cited by-同舟云学术

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Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4

Published:2024-03 Issue:1 Volume:35 Page:102148
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Sai Hali,Ollington Bethany,Rezek Farah O.,Chai Niuzheng,Lane Amelia,Georgiadis Anastasios,Bainbridge James,Michaelides Michel,Sacristan-Reviriego Almudena,Perdigão Pedro R.L.,Leung Amy,van der Spuy Jacqueline^ORCID

Funder

Medical Research Council

Rosetrees Trust

Publisher

Elsevier BV

Reference43 articles.

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

2. The Phenotype of Leber Congenital Amaurosis in Patients With AIPL1 Mutations;Dharmaraj;Arch. Ophthalmol.,2004

3. Mutations in a new photoreceptor-pineal gene on 17p cause Leber congenital amaurosis;Sohocki;Nat. Genet.,2000

4. The Leber congenital amaurosis gene product AIPL1 is localized exclusively in rod photoreceptors of the adult human retina;van der Spuy;Hum. Mol. Genet.,2002

5. The expression of the Leber congenital amaurosis protein AIPL1 coincides with rod and cone photoreceptor development;van der Spuy;Invest. Ophthalmol. Vis. Sci.,2003

Cited by 1 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Application of eye organoids in the study of eye diseases;Experimental Eye Research;2024-10

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