- AVB-101 is an investigational one-time therapy designed to deliver a functional copy of the GRN gene directly to the brain, thereby potentially restoring progranulin levels and stopping disease ...
Side-by-side image shows control neurons from the study (left) compared to neurons from the study with the mis-spliced KCNQ2 gene. Protein TDP-43 malfunctions and disrupts the normal splicing of the ...
Neuroscientists at Macquarie University in Australia have developed a single-dose genetic medicine that has been proven to halt the progression of both ALS and frontotemporal dementia (FTD) in mice – ...
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