Identification of 53 compounds that block Ebola virus-like particle entry via a repurposing screen of approved drugs
- Jennifer Kouznetsova, Wei Sun, A. García-Sastre
- BiologyEmerging Microbes and Infections
- 1 January 2014
The identification of 53 drugs with activity of blocking Ebola VLP entry into cells are reported, including microtubule inhibitors, estrogen receptor modulators, antihistamines, antipsychotics, pump/channel antagonists, and anticancer/antibiotics.
Phenotypic screens as a renewed approach for drug discovery.
- Wei Zheng, Natasha Thorne, J. McKew
- Biology, ChemistryDrug Discovery Today
- 1 November 2013
Targeting Acyl-CoA:Diacylglycerol Acyltransferase 1 (DGAT1) with Small Molecule Inhibitors for the Treatment of Metabolic Diseases
- Jingsong Cao, Yingjiang Zhou, R. Gimeno
- Biology, ChemistryJournal of Biological Chemistry
- 11 October 2011
T-863, a potent DGAT1 inhibitor acting on the acyl-CoA binding site of DGat1, decreased body weight, improved insulin sensitivity, and alleviated hepatic steatosis in diet-induced obese mice, and suggests that pharmacological inhibition of DG AT1 holds promise in treating diverse metabolic disorders.
Chemical signatures and new drug targets for gametocytocidal drug development
- Wei Sun, Takeshi Q. Tanaka, Wei Zheng
- BiologyScientific Reports
- 17 January 2014
The identification of 27 potent gametocytocidal compounds from screening 5,215 known drugs and compounds provide critical new leads and potential targets to expand the repertoire of malaria transmission-blocking reagents.
Mutation Update for GNE Gene Variants Associated with GNE Myopathy
- F. Celeste, T. Vilboux, M. Huizing
- BiologyHuman Mutation
- 1 August 2014
The GNE gene encodes the rate‐limiting, bifunctional enzyme of sialic acid biosynthesis, uridine diphosphate‐N‐acetylglucosamine 2‐epimerase/N-acetylmannosamine kinase (GNE), an adult‐onset progressive myopathy, and three frequently occurring GNE missense variants/polymorphisms are identified, important for future sequence interpretations.
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
- D. Ory, E. Ottinger, F. Porter
- MedicineThe Lancet
- 14 October 2017
Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease.
- E. Ottinger, M. Kao, J. McKew
- BiologyCurrent Topics in Medicinal Chemistry
- 31 January 2014
How this collaborative effort helped to overcome scientific, clinical and financial challenges facing the development of new drug treatments for rare and neglected diseases, and how it will incentivize the commercialization of HP-β-CD for the benefit of the NPC patient community are discussed are discussed.
Blockade of cytosolic phospholipase A2α prevents experimental autoimmune encephalomyelitis and diminishes development of Th1 and Th17 responses
- S. Marusic, P. Thakker, J. Clark
- BiologyJournal of Neuroimmunology
- 15 November 2008
δ-Tocopherol Reduces Lipid Accumulation in Niemann-Pick Type C1 and Wolman Cholesterol Storage Disorders*
The data suggest that regulated exocytosis may represent a potential therapeutic target for reduction of lysosomal storage in this class of diseases.
Small molecule inhibition of group I p21-activated kinases in breast cancer induces apoptosis and potentiates the activity of microtubule stabilizing agents
- C. Ong, S. Gierke, K. Hoeflich
- Biology, MedicineBreast Cancer Research
- 23 April 2015
Findings further support PAK1 as a potential target in breast cancer and suggest combination with taxanes as a viable strategy to increase anti-tumor efficacy.