Post Type Description
Gene-based therapeutics, including gene delivery and RNA interference (RNAi)-based therapies, have made tremendous advances over the past several years towards the development of therapeutics for ALS/FTD and numerous other diseases. The objective of this application is to develop potential therapeutics…
A mutation in a gene called C9orf72 is the most common genetic cause of ALS. In our initial work in fruit flies and in human nerve cells, we found that the C9orf72 mutation changes the levels of specific molecules called…
The Coleman Lab is investigating a protein, SARM1, whose removal strongly protects nerve cells. Rare mutations that hyperactivate SARM1 are enriched in ALS, while other data suggest wider roles in ALS making it an important drug target. Our data indicate…
The Bosco laboratory is studying stress as an upstream trigger of neuron death in ALS. These studies are based on observations that neurons in ALS patients become overactivated because of altered neuronal connections and metabolite imbalances within the central nervous…
Motor neurons are the cells affected in ALS. In most cases, group of motor neurons controlling eye movements are spared. It is unclear why some motor neurons are selectively spared in ALS. Microglia, the resident immune cells of the brain,…
The Mohassel Lab recently discovered a new genetic form of ALS that stems from excessive activity of an enzyme called serine palmitoyltransferase. This study seeks to create cellular and animal models of a new form of ALS to enable future…
The protein NUP50 is decreased in ALS. This project will seek to understand the importance of NUP50 decrease in motor neurons to define the role of this protein in the disease.
This project aims to test IL-18 receptor function in human C9orf72 models of ALS and to test IL-18 pathway inhibitors as therapeutic for patients with ALS based on recent genetics discoveries from the Hornstein lab at Weizmann Institute of Science.
A major hallmark of ALS pathology is incorrect localisation of a protein called TDP-43. When in the incorrect location TDP-43 can initiate degeneration of brain cells. This project aims to dissect whether this toxicity depends on three different capabilities of…
Dysregulation of RNA metabolism is a defining feature of ALS, resulting from the nuclear clearance of the RNA-binding protein TDP-43. TDP-43 is known to bind to its target pre-mRNAs near polyadenylation signals; however, this key aspect of TDP-43 function has…
