Researchers from Macquarie University have detailed the discovery of a novel gene therapy vector targeting pathological TAR-binding protein 43 (TDP-43), CTx-1000, as a potential therapeutic candidate for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) – two diseases characterized by cytoplasmic deposition of the nuclear TDP-43.
Several neurodegenerative disorders have TAR DNA-binding protein 43 (TDP-43) inclusions as a pathological hallmark; thus, the development of PET tracers able to detect TDP-43 aggregates is essential to advance the diagnosis and treatment monitoring in diseases such as frontotemporal dementia, amyotrophic lateral sclerosis and others.
The suppression of the SYF2 factor could be a new therapeutic strategy for the treatment of the different types of amyotrophic lateral sclerosis (ALS). According to a study from the University of Southern California, SYF2 acts on the TDP-43 protein, improving the survival of motor neurons affected by this disease. “We wanted to find something that would improve neuron survival across many different iPSC lines for ALS,” Justin Ichida told BioWorld.
Target ALS Foundation Inc. and the Alzheimer's Drug Discovery Foundation LLC (ADDF) have announced the first four award recipients in a new initiative to identify and develop biomarkers for neurodegenerative diseases, including Alzheimer's disease, amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD).
Australian researchers are the first to discover how inflammation is triggered in motor neuron disease (MND) and identified the molecules involved, which could be the first step toward development of treatments to slow the progression of MND and possibly other debilitating neurological diseases.