High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models

Friedreich's Ataxia: Autosomal Recessive Disease Caused by an Intronic GAA Triplet Repeat Expansion

Victoria Campuzano, Laura Montermini, María Dolores Moltó et al. · Science · 1996 · 2.7K citations

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Catherine S. Manno, Glenn F. Pierce, Valder R. Arruda et al. · Nature Medicine · 2006 · 2.1K citations

Inflammation, Aav-factor Ix, Host Response +11

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 et al. · Human Gene Therapy · 2018 · 757 citations · Full text

Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

Joseph E. Rabinowitz, Fabienne Rolling, Chengwen Li et al. · Journal of Virology · 2002 · 743 citations · Full text

Mouse models for Friedreich ataxia exhibit cardiomyopathy, sensory nerve defect and Fe-S enzyme deficiency followed by intramitochondrial iron deposits

Hélène Puccio, Delphine Simon, Mireille Cossée et al. · Nature Genetics · 2001 · 729 citations

Intramitochondrial Iron Deposits, Sensory Nerve Defect, Iron Metabolism +9