Developing Topics.

Background: The double-blind (DB) period of the DIAN-TU-001 phase 3 trial with gantenerumab provided evidence of significant but incomplete reduction of amyloid plaques, cerebrospinal fluid total tau, and phospho-tau181 in dominantly inherited Alzheimer's disease (DIAD).Subsequently, eligible participants transitioned to an open-label extension (OLE) period using higher doses of gantenerumab (1500mg SC-administered every two weeks [q2w]).

Method: 73 DIAD participants entered the OLE period.

Long-term safety of gantenerumab in participants with Alzheimer's disease: A phase III, open-label extension study (SCarlet RoAD).

Background: Gantenerumab is a fully human anti-amyloid-β (Aβ) immunoglobulin G1 monoclonal antibody for subcutaneous (SC) administration. The efficacy and safety of low-dose (105 mg or 225 mg) gantenerumab were investigated in SCarlet RoAD (SR; NCT01224106), a Phase III, double-blind (DB), placebo-controlled study in participants with prodromal Alzheimer's disease. Following a pre-planned futility analysis, SR was converted into an open-label extension (OLE) study.

Shared and unique consequences of Joubert Syndrome gene dysfunction on the zebrafish central nervous system.

Joubert Syndrome (JBTS) is a neurodevelopmental ciliopathy defined by a highly specific midbrain-hindbrain malformation, variably associated with additional neurological features. JBTS displays prominent genetic heterogeneity with >40 causative genes that encode proteins localising to the primary cilium, a sensory organelle that is essential for transduction of signalling pathways during neurodevelopment, among other vital functions. JBTS proteins localise to distinct ciliary subcompartments, suggesting diverse functions in cilium biology.

Automation of biochemical assays using an open-sourced, inexpensive robotic liquid handler.

High Throughput Screening is crucial in pharmaceutical companies for efficient testing in drug discovery and development. Our Vaccines Analytical Research and Development (V-AR&D) department extensively uses robotic liquid handlers in their High Throughput Analytics (HTA) group for assay development and sample screening. However, these instruments are expensive and require extensive training.

Transient Inhibition of Translation Improves Cardiac Function After Ischemia/Reperfusion by Attenuating the Inflammatory Response.

Background: The myocardium adapts to ischemia/reperfusion (I/R) by changes in gene expression, determining the cardiac response to reperfusion. mRNA translation is a key component of gene expression. It is largely unknown how regulation of mRNA translation contributes to cardiac gene expression and inflammation in response to reperfusion and whether it can be targeted to mitigate I/R injury.