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New York, NY 10010
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Publications

Friedreich’s Ataxia

Expression and processing of mature human frataxin after gene therapy in mice

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Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia

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Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy

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Correction of half the cardiomyocytes fully rescue Friedreich ataxia mitochondrial cardiomyopathy through cell-autonomous mechanisms

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Arrhythmogenic Cardiomyopathy

Plakophilin 2 gene therapy prevents and rescues arrhythmogenic right ventricular cardiomyopathy in a mouse model harboring patient genetics

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Plakophilin-2 Gene Therapy Prevents Arrhythmogenic Right Ventricular Cardiomyopathy Development In A Novel Mouse Model Harboring Patient Genetics

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Diagnostic and therapeutic strategies for arrhythmogenic right ventricular dysplasia/cardiomyopathy patient

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Truncating plakophilin-2 mutations in arrhythmogenic cardiomyopathy are associated with protein haploinsufficiency in both myocardium and epidermis

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Molecular Insights into Arrhythmogenic Right Ventricular Cardiomyopathy Caused by Plakophilin-2 Missense Mutations

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APOE4 Associated Alzheimer’s

APOE4/4 is linked to damaging lipid droplets in Alzheimer’s disease microglia

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APOE3ch alters microglial response and suppresses Aβ-induced tau seeding and spread

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APOE in the bullseye of neurodegenerative diseases: impact of the APOE genotype in Alzheimer's disease pathology and brain diseases

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Resistance to autosomal dominant Alzheimer’s disease in an APOE3 Christchurch homozygote: a case report

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Apolipoprotein E and Alzheimer disease: pathobiology and targeting strategies

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AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

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Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models

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Vector Serotypes

Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates (December 2020)

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