New in vitro models to study amyotrophic lateral sclerosis.
Sheffield, United Kingdom. In Brain Pathol, Feb 2016
The identification of mutations in transactive response DNA-binding protein gene (TARDBP), fused in sarcoma (FUS) and, more recently, a GGGGCC-hexanucleotide repeat expansion in chromosome 9 open reading frame 72 (C9ORF72) and their link with familial ALS have provided new avenues of investigation and hypotheses on the pathophysiology of this devastating disease.
Drug and gene delivery across the blood-brain barrier with focused ultrasound.
Charlottesville, United States. In J Control Release, Jan 2016
It is now well-established that focused ultrasound (FUS) in conjunction with contrast agent microbubbles may be used to non-invasively and temporarily disrupt the BBB, allowing localized delivery of systemically administered therapeutic agents as large as 100nm in size to the CNS.