Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

Christian Hinderer, Nathan Katz, Elizabeth L. Buza, +5

Human Gene Therapy

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang Yang, Lili Wang, Peter Bell, +9

Nature Biotechnology

Systems BiologyHepatologyCas9-mediated CorrectionLiver PhysiologyInherited Metabolic Disease +8

CpG-depleted adeno-associated virus vectors evade immune detection

Susan M. Faust, Peter Bell, Benjamin J. Cutler, +4

Journal of Clinical Investigation

Skeletal Muscle DiseasesAdaptive Immune SystemAav VectorImmunologyImmune Regulation +18

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

Luk H. Vandenberghe, Peter Bell, Albert M. Maguire, +10

Science Translational Medicine

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier

Juliette Hordeaux, Yuan Yuan, Peter Clark, +7

Molecular Therapy

Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors

Lili Wang, Roberto Calcedo, Peter Bell, +4

Human Gene Therapy

Aav8 Gene TransferViral PathogenesisImmunologyPathologyAav8 Vector +15

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype

Rita Sarkar, Renee Tetreault, Guangping Gao, +5

Blood

The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice

Christie Bell, Luk H. Vandenberghe, Peter Bell, +5

Journal of Clinical Investigation

Lung InflammationImmunologyGlycobiologyViral PathogenesisViral Structural Protein +15

Biology of AAV Serotype Vectors in Liver-Directed Gene Transfer to Nonhuman Primates

Guangping Gao, You Lü, Roberto Calcedo, +6

Molecular Therapy

MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates

Juliette Hordeaux, Elizabeth L. Buza, Brianne A Jeffrey, +9

Science Translational Medicine