Relevant Publications
Friedreich’s Ataxia
Expression and processing of mature human frataxin after gene therapy in mice
PublicationIdentification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia
PublicationStress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy
PublicationCorrection of half the cardiomyocytes fully rescue Friedreich ataxia mitochondrial cardiomyopathy through cell-autonomous mechanisms
PublicationA 22-Year Follow-up Study of Long-term Cardiac Outcome and Predictors of Survival in Friedreich Ataxia
PublicationArrhythmogenic Cardiomyopathy
Plakophilin 2 gene therapy prevents and rescues arrhythmogenic right ventricular cardiomyopathy in a mouse model harboring patient genetics
PublicationPlakophilin-2 Gene Therapy Prevents Arrhythmogenic Right Ventricular Cardiomyopathy Development In A Novel Mouse Model Harboring Patient Genetics
PublicationDiagnostic and therapeutic strategies for arrhythmogenic right ventricular dysplasia/cardiomyopathy patient
PublicationTruncating plakophilin-2 mutations in arrhythmogenic cardiomyopathy are associated with protein haploinsufficiency in both myocardium and epidermis
PublicationMolecular Insights into Arrhythmogenic Right Ventricular Cardiomyopathy Caused by Plakophilin-2 Missense Mutations
PublicationAPOE4 Associated Alzheimer’s
AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in Alzheimer’s disease mice
PublicationAPOE4 homozygozity represents a distinct genetic form of Alzheimer’s disease
PublicationAPOE4/4 is linked to damaging lipid droplets in Alzheimer’s disease microglia
PublicationAPOE3ch alters microglial response and suppresses Aβ-induced tau seeding and spread
PublicationAPOE in the bullseye of neurodegenerative diseases: impact of the APOE genotype in Alzheimer's disease pathology and brain diseases
PublicationResistance to autosomal dominant Alzheimer’s disease in an APOE3 Christchurch homozygote: a case report
PublicationApolipoprotein E and Alzheimer disease: pathobiology and targeting strategies
PublicationAAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease
PublicationIntracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models
PublicationVector Serotypes
Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates (December 2020)
PublicationChemistry Manufacturing and Controls
Improving VP1 Ratios Impact on CQAs in rh10 AAV Manufactured through Sf9 Platform
PublicationDevelopment of a Novel High-Yielding Scalable Sf9-Baculovirus Platform to Produce Quality AAV at 200L Scale
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