AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice

Gene Action in the X-chromosome of the Mouse (Mus musculus L.)

Mary F. Lyon · Nature · 1961 · 4K citations

Life Sciences, Genetics, Molecular Genetics +12

Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

Martin Lock, Mauricio R. Alvira, Luk H. Vandenberghe et al. · Human Gene Therapy · 2010 · 334 citations · Full text

Life Sciences, Biotechnology, Molecular Biology +15

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

Andrew M. Davidoff, Catherine Y. Ng, Junfang Zhou et al. · Blood · 2003 · 240 citations · Full text

Life Sciences, Endocrinology, Molecular Biology +16

Cross-sectional multicenter study of patients with urea cycle disorders in the United States

Mendel Tuchman, Brendan Lee, Uta Lichter‐Konecki et al. · Molecular Genetics and Metabolism · 2008 · 218 citations · Full text

Life Sciences, Nephrology, Pediatric Nephrology +4

Prolonged Metabolic Correction in Adult Ornithine Transcarbamylase-deficient Mice with Adenoviral Vectors

Xuehai Ye, Michael B. Robinson, Mark L. Batshaw et al. · Journal of Biological Chemistry · 1996 · 154 citations · Full text

Life Sciences, Biochemistry, Molecular Biology +17