Background Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We
investigated the use of a new gene therapy in patients with the disorder. Methods We infused a …

Background In patients with severe hemophilia B, gene therapy that is mediated by a novel
self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to …

Background The Pediatric Preclinical Testing Program (PPTP) is an initiative supported by
the National Cancer Institute (NCI) to identify novel therapeutic agents that may have …

Heterotransplantation of human cancer cells or tumor biopsies into immunodeficient rodents
(xenograft models) has, for the past two decades, constituted the major preclinical screen …

Recombinant adeno-associated virus (rAAV) vectors encoding human factor VIII (hFVIII)
were systematically evaluated for hemophilia A (HA) gene therapy. A 5.7-kb rAAV-expression …

Background MLN8237 is a small molecule inhibitor of Aurora Kinase A (AURKA) that is
currently in early phase clinical testing. AURKA plays a pivotal role in centrosome maturation …

We present the first crystal structures of a human protein bound to analogs of cocaine and
heroin. Human carboxylesterase 1 (hCE1) is a broad-spectrum bioscavenger that catalyzes …

Carboxylesterases (CE) are ubiquitous enzymes responsible for the metabolism of xenobiotics.
Because the structural and amino acid homology among esterases of different classes, …

Background Rapamycin is a highly specific inhibitor of mTOR, a serine/threonine kinase that
controls cap‐dependent translation. Here we report the activity of rapamycin against the in …

Background SCH 717454 (19D12) is a fully human antibody directed against the insulin‐like
growth factor 1 receptor (IGF‐1R), which is implicated in the growth and metastatic …