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Research article

First published 2025

Discovery of rare and novel variants in inherited retinal disorders: Exome sequencing analysis of six Iranian families

Jafar Aghajani, Abbas Doosti, Mojtaba Jafarinia [...]

Journal of Investigative Medicine

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Review article

First published 2019-9

An Update on Ocular Gene Therapy for Monogenic and Multifactorial Retinal Diseases

Alice Y. Hou, Szilárd Kiss

Journal of VitreoRetinal Diseases

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Research article

First published 2013-06

Human Induced Pluripotent Stem Cells As a Tool to Model a Form of Leber Congenital Amaurosis

Céline Lustremant, Walter Habeler, Alexandra Plancheron [...]

Cellular Reprogramming

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Review article

First published 2018-7

Gene Therapy for Inherited Retinopathies: Update on Development Progress

Susan Sun, Sandra R. Montezuma

Journal of VitreoRetinal Diseases

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Research article

First published 2014-12

Genome-Wide Homozygosity Mapping in Families with Leber Congenital Amaurosis Identifies Mutations in AIPL1 and RDH12 Genes

Didem Yücel-Yılmaz, Berçin Tarlan, Hayyam Kıratlı [...]

DNA and Cell Biology

Open access

Research article

First published 2011-10

Long-Term Preservation of Cones and Improvement in Visual Function Following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Caused by Guanylate Cyclase-1 Deficiency

Marija Mihelec, Rachael A. Pearson, Scott J. Robbie [...]

Human Gene Therapy

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Research article

First published 2013-10

Successful Gene Therapy in Older Rpe65-Deficient Dogs Following Subretinal Injection of an Adeno-Associated Vector Expressing RPE65

Matthew J. Annear, Freya M. Mowat, Joshua T. Bartoe [...]

Human Gene Therapy

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Research article

First published 2010-08

Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis

Basil S. Pawlyk, Oleg V. Bulgakov, Xiaoqing Liu [...]

Human Gene Therapy

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Research article

First published 2013-02

AAV-Mediated Gene Therapy in the Guanylate Cyclase (RetGC1/RetGC2) Double Knockout Mouse Model of Leber Congenital Amaurosis

Sanford L. Boye, Igor V. Peshenko, Wei Chieh Huang [...]

Human Gene Therapy

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Review article

First published 2010-08

Ocular Gene Therapy: An Evaluation of Recombinant Adeno-Associated Virus-Mediated Gene Therapy Interventions for the Treatment of Ocular Disease

Kamolika Roy, Linda Stein, Shalesh Kaushal

Human Gene Therapy

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