Dual ABCA4-AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease-Reference-Cited by-同舟云学术

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Dual ABCA4-AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease

Published:2019-11-01 Issue:11 Volume:30 Page:1361-1370
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Dyka Frank M.¹,Molday Laurie L.²,Chiodo Vince A.¹,Molday Robert S.²³,Hauswirth William W.¹

Affiliation:

1. Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, Florida

2. Department of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, British Columbia, Canada.

3. Department of Ophthalmology and Visual Sciences, University of British Columbia, Vancouver, British Columbia, Canada.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.132

Reference65 articles.

1. Correction: Corrigendum: A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy

2. Stargardt's ABCR is localized to the disc membrane of retinal rod outer segments

3. Expression of ABCA4 in the retinal pigment epithelium and its implications for Stargardt macular degeneration

4. The 220-kDa Rim Protein of Retinal Rod Outer Segments Is a Member of the ABC Transporter Superfamily

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1. The ABCs of Stargardt disease: the latest advances in precision medicine;Cell & Bioscience;2024-07-26

2. Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD;Gene Therapy;2024-05-16

3. Delivering large genes using adeno-associated virus and the CRE-lox DNA recombination system;2024-04-10

4. Allele-specific antisense oligonucleotides for the treatment of BEST1-related dominantly inherited retinal diseases: An in vitro model;Experimental Eye Research;2024-04

5. Cell-cell interaction in the pathogenesis of inherited retinal diseases;Frontiers in Cell and Developmental Biology;2024-03-04

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