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Pathway-based analysis of genome-wide siRNA screens reveals the regulatory landscape of APP processing

PloS one, 2015-02, Vol.10 (2), p.e0115369-e0115369

2015

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Presenilin-Dependent γ-Secretase Activity Modulates Thymocyte Development

Proceedings of the National Academy of Sciences - PNAS, 2001-07, Vol.98 (16), p.9312-9317

2001

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C‐terminal PAL motif of presenilin and presenilin homologues required for normal active site conformation

Journal of neurochemistry, 2006-01, Vol.96 (1), p.218-227

2006

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Correction: Pathway-Based Analysis of Genome-Wide siRNA Screens Reveals the Regulatory Landscape of App Processing

PloS one, 2015-06, Vol.10 (6), p.e0129641-e0129641

2015

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Ocular Inflammatory Response to Intravitreal Injection of Adeno-Associated Virus Vector: Relative Contribution of Genome and Capsid

Human gene therapy, 2020-01, Vol.31 (1-2), p.80-89

2020

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A highly efficient transgene knock-in technology in clinically relevant cell types

Nature biotechnology, 2024-03, Vol.42 (3), p.458-469

2024

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Increased O-GlcNAcylation reduces pathological tau without affecting its normal phosphorylation in a mouse model of tauopathy

Neuropharmacology, 2014-04, Vol.79, p.307-313

2014

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The Impact of Inherited Retinal Diseases in the United States of America (US) and Canada from a Cost-of-Illness Perspective

Clinical ophthalmology (Auckland, N.Z.), 2021-01, Vol.15, p.2855-2866

2021

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Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

Human gene therapy, 2020-07, Vol.31 (13-14), p.743-755

2020

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Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

Molecular therapy, 2017-08, Vol.25 (8), p.1866-1880

2017

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Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

Human gene therapy, 2020-02, Vol.31 (3-4), p.253-267

2020

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Fenton chemistry and oxidative stress mediate the toxicity of the β-amyloid peptide in a Drosophila model of Alzheimer's disease

The European journal of neuroscience, 2009-04, Vol.29 (7), p.1335-1347

2009

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The Intramembrane Cleavage Site of the Amyloid Precursor Protein Depends on the Length of Its Transmembrane Domain

Proceedings of the National Academy of Sciences - PNAS, 2002-02, Vol.99 (3), p.1365-1370

2002

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Glu-333 of Nicastrin Directly Participates in γ-Secretase Activity

The Journal of biological chemistry, 2009-10, Vol.284 (43), p.29714-29724

2009

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Selected non-steroidal anti-inflammatory drugs and their derivatives target gamma-secretase at a novel site. Evidence for an allosteric mechanism

The Journal of biological chemistry, 2004-10, Vol.279 (42), p.43419-43426

2004

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Photoactivated γ-secretase inhibitors directed to the active site covalently label presenilin 1

Nature (London), 2000-06, Vol.405 (6787), p.689-694

2000

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Oral treatment with a gamma-secretase inhibitor improves long-term potentiation in a mouse model of Alzheimer's disease

The Journal of pharmacology and experimental therapeutics, 2010-04, Vol.333 (1), p.110-119

2010

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Acute gamma-secretase inhibition of nonhuman primate CNS shifts amyloid precursor protein (APP) metabolism from amyloid-beta production to alternative APP fragments without amyloid-beta rebound

The Journal of neuroscience, 2010-05, Vol.30 (19), p.6743-6750

2010

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L-685,458, an Aspartyl Protease Transition State Mimic, Is a Potent Inhibitor of Amyloid β-Protein Precursor γ-Secretase Activity

Biochemistry (Easton), 2000-08, Vol.39 (30), p.8698-8704

2000

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Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep

Human gene therapy. Clinical development, 2017-06, Vol.28 (2), p.96-107

2017

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The novel gamma secretase inhibitor N-[cis-4-[(4-chlorophenyl)sulfonyl]-4-(2,5-difluorophenyl)cyclohexyl]-1,1,1-trifluoromethanesulfonamide (MRK-560) reduces amyloid plaque deposition without evidence of notch-related pathology in the Tg2576 mouse

The Journal of pharmacology and experimental therapeutics, 2007-02, Vol.320 (2), p.552-558

2007

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Potent benzoazepinone γ-secretase modulators with improved bioavailability

Bioorganic & medicinal chemistry letters, 2015-09, Vol.25 (17), p.3495-3500

2015

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γ-Secretase Complex Assembly within the Early Secretory Pathway

The Journal of biological chemistry, 2005-02, Vol.280 (8), p.6471-6478

2005

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Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice

Human gene therapy. Clinical development, 2018-12, Vol.29 (4), p.188-197

2018

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Discovery of novel triazolobenzazepinones as γ-secretase modulators with central Aβ42 lowering in rodents and rhesus monkeys

Bioorganic & medicinal chemistry letters, 2015-09, Vol.25 (17), p.3488-3494

2015

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Pathway-based analysis of genome-wide siRNA screens reveals the regulatory landscape of APP processing

Presenilin-Dependent γ-Secretase Activity Modulates Thymocyte Development

C‐terminal PAL motif of presenilin and presenilin homologues required for normal active site conformation

Correction: Pathway-Based Analysis of Genome-Wide siRNA Screens Reveals the Regulatory Landscape of App Processing

Ocular Inflammatory Response to Intravitreal Injection of Adeno-Associated Virus Vector: Relative Contribution of Genome and Capsid

A highly efficient transgene knock-in technology in clinically relevant cell types

Increased O-GlcNAcylation reduces pathological tau without affecting its normal phosphorylation in a mouse model of tauopathy

The Impact of Inherited Retinal Diseases in the United States of America (US) and Canada from a Cost-of-Illness Perspective

Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

Fenton chemistry and oxidative stress mediate the toxicity of the β-amyloid peptide in a Drosophila model of Alzheimer's disease

The Intramembrane Cleavage Site of the Amyloid Precursor Protein Depends on the Length of Its Transmembrane Domain

Glu-333 of Nicastrin Directly Participates in γ-Secretase Activity

Selected non-steroidal anti-inflammatory drugs and their derivatives target gamma-secretase at a novel site. Evidence for an allosteric mechanism

Photoactivated γ-secretase inhibitors directed to the active site covalently label presenilin 1

Oral treatment with a gamma-secretase inhibitor improves long-term potentiation in a mouse model of Alzheimer's disease

Acute gamma-secretase inhibition of nonhuman primate CNS shifts amyloid precursor protein (APP) metabolism from amyloid-beta production to alternative APP fragments without amyloid-beta rebound

L-685,458, an Aspartyl Protease Transition State Mimic, Is a Potent Inhibitor of Amyloid β-Protein Precursor γ-Secretase Activity

Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep

The novel gamma secretase inhibitor N-[cis-4-[(4-chlorophenyl)sulfonyl]-4-(2,5-difluorophenyl)cyclohexyl]-1,1,1-trifluoromethanesulfonamide (MRK-560) reduces amyloid plaque deposition without evidence of notch-related pathology in the Tg2576 mouse

Potent benzoazepinone γ-secretase modulators with improved bioavailability

γ-Secretase Complex Assembly within the Early Secretory Pathway

Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice

Discovery of novel triazolobenzazepinones as γ-secretase modulators with central Aβ42 lowering in rodents and rhesus monkeys

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