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Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

Proceedings of the National Academy of Sciences - PNAS, 2019-03, Vol.116 (10), p.4496-4501

2019

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

Human gene therapy, 2019-11, Vol.30 (11), p.1361

2019

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Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness

Human molecular genetics, 2015-11, Vol.24 (21), p.6229-6239

2015

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Distinct Phenotypic Consequences of Pathogenic Mutants Associated with Late-Onset Retinal Degeneration

The American journal of pathology, 2023-11, Vol.193 (11), p.1706-1720

2023

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Clarin‐1 expression in adult mouse and human retina highlights a role of Müller glia in Usher syndrome

The Journal of pathology, 2020-02, Vol.250 (2), p.195-204

2020

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AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy

PloS one, 2016-02, Vol.11 (2), p.e0148874-e0148874

2016

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Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

Human gene therapy, 2015-09, Vol.26 (9), p.593

2015

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Expression of red/green-cone opsin mutants K82E, P187S, M273K result in unique pathobiological perturbations to cone structure and function

Frontiers in neuroscience, 2024-02, Vol.18, p.1368089-1368089

2024

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Pathological Effects of Mutant C1QTNF5 (S163R) Expression in Murine Retinal Pigment Epithelium

Investigative ophthalmology & visual science, 2015-10, Vol.56 (11), p.6971-6980

2015

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Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A

Human gene therapy. Part B. Methods, 2014-04, Vol.25 (2), p.166

2014

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Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse

Investigative ophthalmology & visual science, 2011-09, Vol.52 (10), p.7098-7108

2011

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Cholesterol transport via ABCA1: New insights from solid-phase binding assay

Biochimie, 2013-04, Vol.95 (4), p.957-961

2013

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AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis

Human gene therapy, 2013-02, Vol.24 (2), p.189

2013

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Overexpression of Nfe2l1 increases proteasome activity and delays vision loss in a preclinical model of human blindness

Science advances, 2023-07, Vol.9 (28), p.eadd5479-eadd5479

2023

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Co-Expression of Wild-Type and Mutant S163R C1QTNF5 in Retinal Pigment Epithelium

Advances in experimental medicine and biology, 2018, Vol.1074, p.61-66

2018

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Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors

PloS one, 2013-09, Vol.8 (9)

2013

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Relation of Response to Treatment with Dorzolamide in X-Linked Retinoschisis to the Mechanism of Functional Loss in Retinoschisin

American journal of ophthalmology, 2009-01, Vol.147 (1), p.111-115.e1

2009

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Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors

Molecular therapy. Methods & clinical development, 2014-01, Vol.1 (C), p.5-5, Article 5

2014

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Coexpression and Interaction of Wild-type and Missense RS1 Mutants Associated with X-Linked Retinoschisis: Its Relevance to Gene Therapy

Investigative ophthalmology & visual science, 2007-06, Vol.48 (6), p.2491-2497

2007

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Metabotropic glutamate receptors are differentially regulated under elevated intraocular pressure

Journal of neurochemistry, 2004-07, Vol.90 (1), p.190-202

2004

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Transcription of glycolytic genes and operons in Bacillus subtilis: evidence for the presence of multiple levels of control of the gapA operon

Molecular microbiology, 2001-07, Vol.41 (2), p.409-422

2001

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Characterization and Purification of the Discoidin Domain-Containing Protein Retinoschisin and Its Interaction with Galactose

Biochemistry (Easton), 2008-09, Vol.47 (35), p.9098-9106

2008

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Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

Gene therapy, 2013-08, Vol.20 (8), p.824-833

2013

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Subunits of the epithelial sodium channel family are differentially expressed in the retina of mice with ocular hypertension

Journal of neurochemistry, 2005-07, Vol.94 (1), p.120-128

2005

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Disease mechanisms of X‐linked cone dystrophy caused by missense mutations in the red and green cone opsins

The FASEB journal, 2021-10, Vol.35 (10), p.e21927-n/a

2021

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Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

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

Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness

Distinct Phenotypic Consequences of Pathogenic Mutants Associated with Late-Onset Retinal Degeneration

Clarin‐1 expression in adult mouse and human retina highlights a role of Müller glia in Usher syndrome

AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy

Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

Expression of red/green-cone opsin mutants K82E, P187S, M273K result in unique pathobiological perturbations to cone structure and function

Pathological Effects of Mutant C1QTNF5 (S163R) Expression in Murine Retinal Pigment Epithelium

Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A

Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse

Cholesterol transport via ABCA1: New insights from solid-phase binding assay

AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis

Overexpression of Nfe2l1 increases proteasome activity and delays vision loss in a preclinical model of human blindness

Co-Expression of Wild-Type and Mutant S163R C1QTNF5 in Retinal Pigment Epithelium

Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors

Relation of Response to Treatment with Dorzolamide in X-Linked Retinoschisis to the Mechanism of Functional Loss in Retinoschisin

Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors

Coexpression and Interaction of Wild-type and Missense RS1 Mutants Associated with X-Linked Retinoschisis: Its Relevance to Gene Therapy

Metabotropic glutamate receptors are differentially regulated under elevated intraocular pressure

Transcription of glycolytic genes and operons in Bacillus subtilis: evidence for the presence of multiple levels of control of the gapA operon

Characterization and Purification of the Discoidin Domain-Containing Protein Retinoschisin and Its Interaction with Galactose

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

Subunits of the epithelial sodium channel family are differentially expressed in the retina of mice with ocular hypertension

Disease mechanisms of X‐linked cone dystrophy caused by missense mutations in the red and green cone opsins

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