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Retricted access

Research article

First published 2003-6

Studio RM dell'encefalo di trentacinque bambini affetti da amaurosi congenita di Leber

C. Uggetti, E. Fazzi, S. Signorini [...]

The Neuroradiology Journal

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Case report

First published 2020-10

Leber Congenital Amaurosis With Coats-Like Reaction and 3 Novel Sequence Variants: A Short Report

Mehdi Tavakoli, Amy M. Huang, Linda Cernichiaro-Espinosa [...]

Journal of VitreoRetinal Diseases

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Other

First published 1996-03

Leber's Congenital Amaurosis Associated With Mitochondrial Dysfunction

Manuel Castro-Gago, Elena Pintos-Martínez, Andrés Beiras-Iglesias [...]

Journal of Child Neurology

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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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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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Brief report

First published 2009-09

Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

Artur V. Cideciyan, William W. Hauswirth, Tomas S. Aleman [...]

Human Gene Therapy

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Research article

First published 2006-8

Safety in Nonhuman Primates of Ocular AAV2- RPE65,a Candidate Treatment for Blindness in Leber Congenital Amaurosis

Samuel G. Jacobson, Sanford L. Boye, Tomas S. Aleman [...]

Human Gene Therapy

Open access

Research article

First published 2018-01

Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

Wei Zhang, Linjing Li, Qin Su [...]

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 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

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