Replacing or editing disease-causing mutations holds great promise for treating many human
diseases. Yet, delivering therapeutic genetic modifiers to specific cells in vivo has been …

Recognition of the multiple roles of Hedgehog signaling in cancer has prompted intensive
efforts to develop targeted pathway inhibitors. Leading inhibitors in clinical development act …

Inhibition of the AAA ATPase, p97, was recently shown to be a novel method for targeting
the ubiquitin proteasome system, and CB-5083, a first-in-class inhibitor of p97, has …

Background Adeno-associated virus (AAV) vectors are a promising vehicle for noninvasive
gene delivery to the central nervous system via intravenous infusion. However, naturally …

Circulating tumor cells (CTCs) play a fundamental role in cancer progression. However, in
mice, limited blood volume and the rarity of CTCs in the bloodstream preclude longitudinal, in-…

Glucagon-like peptide-1 (GLP-1) receptor knockout (Glp1r −/− ) mice exhibit impaired hepatic
insulin action. High fat (HF)-fed Glp1r −/− mice exhibit improved, rather than the expected …

Recombinant immunotoxins (RITs) are genetically engineered proteins being developed to
treat cancer. They are composed of an Fv that targets a cancer antigen and a fragment of a …

Protein-based drugs are very active in treating cancer, but their efficacy can be limited by
the formation of neutralizing antidrug antibodies (ADAs). Recombinant immunotoxins are …

Genome editing technologies that install diverse edits can widely enable genetic studies
and new therapeutics. Here we develop click editing, a genome writing platform that couples …

Objectives The purpose of this study was to assess serum concentrations of gabapentin in
cats with chronic kidney disease (CKD) vs clinically healthy cats. Methods Five healthy cats …