Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors-Reference-Cited by-同舟云学术

Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

Published:2009-03-19 Issue:12 Volume:18 Page:2099-2114
ISSN:1460-2083
Container-title:Human Molecular Genetics
language:en
Short-container-title:

Author:

Tan Mei Hong,Smith Alexander J.,Pawlyk Basil,Xu Xiaoyun,Liu Xiaoqing,Bainbridge James B.,Basche Mark,McIntosh Jenny,Tran Hoai Viet,Nathwani Amit,Li Tiansen,Ali Robin R.

Publisher

Oxford University Press (OUP)

Subject

Genetics (clinical),Genetics,Molecular Biology,General Medicine

Link

http://academic.oup.com/hmg/article-pdf/18/12/2099/17246169/ddp133.pdf

Reference40 articles.

1. Leber congenital amaurosis;Perrault;Mol. Genet. Metab.,1999

2. Mental retardation in amaurosis congenita of Leber;Schuil;Neuropediatrics,1998

3. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy;Ali;Nat. Genet.,2000

4. Gene therapy restores vision in a canine model of childhood blindness;Acland;Nat. Genet.,2001

5. Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65−/− knockout mouse eye results in limited rescue;Lai;Genet. Vaccines Ther.,2004

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1. Clinical and Molecular Characterization of AIPL1-Associated Leber Congenital Amaurosis/Early-Onset Severe Retinal Dystrophy;American Journal of Ophthalmology;2024-10

2. Correction to: Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors;Human Molecular Genetics;2024-04-03

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4. Retinal Organoids from an AIPL1 CRISPR/Cas9 Knockout Cell Line Successfully Recapitulate the Molecular Features of LCA4 Disease;International Journal of Molecular Sciences;2023-03-21

5. AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice;Human Molecular Genetics;2022-08-04