Using CRISPR to edit the globin gene in blood stem cells has resulted in the highest level of gene correction achieved to date in preclinical models of sickle cell disease (SCD). To be sure, "highest" is still not particularly high. The study, which was published in the Oct. 12, 2016, issue of Science Translational Medicine, showed successful gene correction in 2 percent to 5 percent of blood stem cells.
Mice with glioblastoma showed increased survival when they were treated with a liver X receptor (LXR) agonist, LXR-623 (Pfizer Inc.), which has undergone phase I testing.
Adding a primate version of the monoclonal antibody Entyvio (vedolizumab, Takeda Pharmaceutical Co. Ltd.), which is approved for the treatment of ulcerative colitis and Crohn's disease, to combination antiretroviral therapy (ART) enabled macaque monkeys to control blood levels of SIV long after treatment with both the antibody and the ART had been discontinued.
By studying drug mechanisms of actions in living cells, researchers have been able to show that the ocular toxicity of inhibitors of the beta-secretase beta-site APP-cleaving enzyme 1 (BACE1), which are in clinical trials for the treatment of Alzheimer's disease (AD), is due to inhibition of the related enzyme Cathepsin D. More broadly, they wrote in their paper, their results "underscore the power of chemical proteomics for discerning mechanisms of drug action."
Researchers were able to reverse memory deficits in a mouse model of Alzheimer's disease by preventing cleavage of the protein tau by the enzyme caspase-2.
Formation of amyloid bodies is a widespread coping mechanism to stress in cells, and should be considered a normal organizational feature, scientists from the University of Miami Miller School of Medicine have argued.
