Long-Term Correction of Ammonia Metabolism and Prolonged Survival in Ornithine Transcarbamylase-Deficient Mice Following Liver-Directed Treatment with Adeno-associated Viral Vectors

Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy.

Yang Yang, Frederick A. Nunes, Klára Berencsi et al. · Proceedings of the National Academy of Sciences · 1994 · 1.6K citations

Life Sciences, Immunology, Molecular Biology +15

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

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

Steven E. Raper, Narendra Chirmule, Frank S. Lee et al. · Molecular Genetics and Metabolism · 2003 · 1.5K citations

Life Sciences, Immunology, Molecular Genetics +14

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

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

Douglas M. McCarty, Haiyan Fu, Paul E. Monahan et al. · Gene Therapy · 2003 · 539 citations

Life Sciences, Molecular Biology, Gene Delivery +13