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Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids

Stem cell reports, 2020-07, Vol.15 (1), p.67-79

2020

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Effective delivery of large genes to the retina by dual AAV vectors

EMBO molecular medicine, 2014-02, Vol.6 (2), p.194-211

2014

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SARM1 Ablation Is Protective and Preserves Spatial Vision in an In Vivo Mouse Model of Retinal Ganglion Cell Degeneration

International journal of molecular sciences, 2022-01, Vol.23 (3), p.1606

2022

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AAV-NDI1 Therapy Provides Significant Benefit to Murine and Cellular Models of Glaucoma

International journal of molecular sciences, 2024-08, Vol.25 (16), p.8876

2024

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Non-photoreceptor Expression of Tulp1 May Contribute to Extensive Retinal Degeneration in Tulp1-/- Mice

Frontiers in neuroscience, 2020-06, Vol.14, p.656-656

2020

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RPE-Directed Gene Therapy Improves Mitochondrial Function in Murine Dry AMD Models

International journal of molecular sciences, 2023-02, Vol.24 (4), p.3847

2023

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Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa

Molecular therapy, 2011-04, Vol.19 (4), p.642-649

2011

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AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice

Molecular therapy. Methods & clinical development, 2022-06, Vol.25, p.236-249

2022

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Novel 199 base pair NEFH promoter drives expression in retinal ganglion cells

Scientific reports, 2020-10, Vol.10 (1), p.16515-16515, Article 16515

2020

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Optimized OPA1 Isoforms 1 and 7 Provide Therapeutic Benefit in Models of Mitochondrial Dysfunction

Frontiers in neuroscience, 2020-11, Vol.14, p.571479-571479

2020

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AAV-Delivered Tulp1 Supplementation Therapy Targeting Photoreceptors Provides Minimal Benefit in Tulp1−/− Retinas

Frontiers in neuroscience, 2020-08, Vol.14

2020

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A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors

Frontiers in neuroscience, 2017-09, Vol.11, p.521-521

2017

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Altered retinal microRNA expression profile in a mouse model of retinitis pigmentosa

Genome biology, 2007-01, Vol.8 (11), p.R248-R248, Article R248

2007

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Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction

Pharmaceutics, 2023-01, Vol.15 (2), p.322

2023

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AAV‐mediated gene therapy improving mitochondrial function provides benefit in age‐related macular degeneration models

Clinical and translational medicine, 2022-08, Vol.12 (8), p.e952-n/a

2022

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Efficacy of codelivery of dual AAV2/5 vectors in the murine retina and hippocampus

Human gene therapy, 2012-08, Vol.23 (8), p.847-858

2012

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A common microRNA signature in mouse models of retinal degeneration

Experimental eye research, 2008-12, Vol.87 (6), p.529-534

2008

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A Mutation-Independent Therapeutic Strategy for Dominant Dystrophic Epidermolysis Bullosa

Journal of investigative dermatology, 2013-12, Vol.133 (12), p.2793-2796

2013

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Toward a Gene Therapy for Dominant Disease: Validation of an RNA Interference-Based Mutation-Independent Approach

Molecular therapy, 2005-09, Vol.12 (3), p.555-561

2005

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AAV-mediated chronic over-expression of SNAP-25 in adult rat dorsal hippocampus impairs memory-associated synaptic plasticity

Journal of neurochemistry, 2010-02, Vol.112 (4), p.991-1004

2010

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Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouse

Molecular therapy. Methods & clinical development, 2015-01, Vol.2, p.15016-15016, Article 15016

2015

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Gene-Based Therapies for Dominant Retinopathies

Current gene therapy, 2010-10, Vol.10 (5), p.381-388

2010

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Toward an elucidation of the molecular genetics of inherited retinal degenerations

Human molecular genetics, 2017-08, Vol.26 (R1), p.R2-R11

2017

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Corrigendum to "Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho-/- mouse"

Molecular therapy. Methods & clinical development, 2016, Vol.3 (C), p.16032-16032, Article 16032

2016

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A novel homozygous truncating GNAT1 mutation implicated in retinal degeneration

British journal of ophthalmology, 2016-04, Vol.100 (4), p.495-500

2016

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Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids

Effective delivery of large genes to the retina by dual AAV vectors

SARM1 Ablation Is Protective and Preserves Spatial Vision in an In Vivo Mouse Model of Retinal Ganglion Cell Degeneration

AAV-NDI1 Therapy Provides Significant Benefit to Murine and Cellular Models of Glaucoma

Non-photoreceptor Expression of Tulp1 May Contribute to Extensive Retinal Degeneration in Tulp1-/- Mice

RPE-Directed Gene Therapy Improves Mitochondrial Function in Murine Dry AMD Models

Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa

AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice

Novel 199 base pair NEFH promoter drives expression in retinal ganglion cells

Optimized OPA1 Isoforms 1 and 7 Provide Therapeutic Benefit in Models of Mitochondrial Dysfunction

AAV-Delivered Tulp1 Supplementation Therapy Targeting Photoreceptors Provides Minimal Benefit in Tulp1−/− Retinas

A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors

Altered retinal microRNA expression profile in a mouse model of retinitis pigmentosa

Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction

AAV‐mediated gene therapy improving mitochondrial function provides benefit in age‐related macular degeneration models

Efficacy of codelivery of dual AAV2/5 vectors in the murine retina and hippocampus

A common microRNA signature in mouse models of retinal degeneration

A Mutation-Independent Therapeutic Strategy for Dominant Dystrophic Epidermolysis Bullosa

Toward a Gene Therapy for Dominant Disease: Validation of an RNA Interference-Based Mutation-Independent Approach

AAV-mediated chronic over-expression of SNAP-25 in adult rat dorsal hippocampus impairs memory-associated synaptic plasticity

Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouse

Gene-Based Therapies for Dominant Retinopathies

Toward an elucidation of the molecular genetics of inherited retinal degenerations

Corrigendum to "Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho-/- mouse"

A novel homozygous truncating GNAT1 mutation implicated in retinal degeneration

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