Hans Gaus

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary<i>N</i>-acetyl galactosamine improves potency 10-fold in mice

Thazha P. Prakash, Mark J. Graham, Jinghua Yu et al. · Nucleic Acids Research · 2014 · 597 citations · Full text

Pharmacy, Molecular Pharmaceutics, Pharmacology +17

Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates

Frank Rigo, Seung Chun, Daniel A. Norris et al. · Journal of Pharmacology and Experimental Therapeutics · 2014 · 275 citations · Full text

Medicine, Neuroscience, Pharmacology +14

Single-Stranded siRNAs Activate RNAi in Animals

Walt F. Lima, Thazha P. Prakash, Heather Murray et al. · Cell · 2012 · 257 citations · Full text

Life Sciences, Genetics, Molecular Biology +10

Cross-Species Pharmacokinetic Comparison from Mouse to Man of a Second-Generation Antisense Oligonucleotide, ISIS 301012, Targeting Human Apolipoprotein B-100

Rosie Z. Yu, Tae‐Won Kim, An Hong et al. · Drug Metabolism and Disposition · 2006 · 238 citations

Pharmacy, Pharmacokinetics, Pharmacology +13

Synthesis and Biophysical Evaluation of 2′,4′-Constrained 2′<i>O</i>-Methoxyethyl and 2′,4′-Constrained 2′<i>O</i>-Ethyl Nucleic Acid Analogues

Punit P. Seth, Guillermo Vasquez, Charles A. Allerson et al. · The Journal of Organic Chemistry · 2010 · 211 citations

Pharmacy, Medicinal Chemistry, Molecular Biology +17