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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 2023-07

Gene Supplementation in Mice Heterozygous for the D477G RPE65 Variant Implicated in Autosomal Dominant Retinitis Pigmentosa

Kecia L. Feathers, Lin Jia, Naheed W. Khan [...]

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

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Research article

First published 2008-10

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

William W. Hauswirth, Tomas S. Aleman, Shalesh Kaushal [...]

Human Gene Therapy

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Research article

First published 2018-12

Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy

Mark E. Pennesi, Richard G. Weleber, Paul Yang [...]

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 2018-10

Effects of fibrin glue as a three-dimensional scaffold in cultivated adult human retinal pigment epithelial cells

Sahar Balagholi, Mozhgan Rezaei Kanavi, Shaban Alizadeh [...]

Journal of Biomaterials Applications

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Research article

First published 2018-02

Improving the Transduction of Bone Marrow–Derived Cells with an Integrase-Defective Lentiviral Vector

S. Louise Pay, Xiaoping Qi, Jeffrey F. Willard [...]

Human Gene Therapy Methods (Part B)

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Research article

First published 2015-12

Investor Outlook: Significance of the Positive LCA2 Gene Therapy Phase III Results

Joshua Schimmer, Steven Breazzano

Human Gene Therapy Clinical Development

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Research article

First published 2010-12

Molecular Anthropology Meets Genetic Medicine to Treat Blindness in the North African Jewish Population: Human Gene Therapy Initiated in Israel

Eyal Banin, Dikla Bandah-Rozenfeld, Alexey Obolensky [...]

Human Gene Therapy

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