Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Guangping Gao, Mauricio R. Alvira, Lili Wang et al. · Proceedings of the National Academy of Sciences · 2002 · 1.5K citations · Full text

Life Sciences, Molecular Biology, Molecular Genetics +15

α-Glucosidase deficiency in generalized glycogen-storage disease (Pompe's disease)

Hers Hg · Biochemical Journal · 1963 · 865 citations · Full text

Life Sciences, Biochemistry, Genetics +12

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 · 750 citations · Full text

Life Sciences, Biotechnology, Molecular Biology +14

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Catherine S. Manno, Ah Keng Chew, S Hutchison et al. · Blood · 2003 · 747 citations · Full text

Medicine, Genetics, Hematology +11

Systemic delivery of genes to striated muscles using adeno-associated viral vectors

Paul Gregorevic, Michael J. Blankinship, James M. Allen et al. · Nature Medicine · 2004 · 611 citations · Full text

Pharmacy, Bioengineering, Molecular Biology +14