The small molecule successfully targeted the C9orf72 gene that causes amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
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frontotemporal dementia (FTD)
Researchers at the University of East Finland have been using skin cells to investigate pathological hallmarks in frontotemporal dementia patients.
How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy
In this article, Dr Jim Burns discusses promising pre-clinical results of how a new platform could treat the root cause of many devastating genetic diseases including myotonic dystrophy type 1.
According to researchers, the more small tau protein variants expressed by neurons, the slower neurofibrillary tangles form.
New findings on dementia reveal that brain atrophy spreads via connected brain networks, rather than simply adjacent areas of the brain.
Study provides a roadmap for using CRISPR to investigate neurological disorders...