It’s ‘And,’ Not ‘Either-Or’: C9ORF72 Mechanisms of Action are Linked Beyond the Nucleus: TDP-43 Sticks Together, For Better or Worse Going the Distance: FUS Travels to Terminals, Drops Off RNA When RNA transcripts aren’t processed properly or don’t make i
At an RNA metabolism meeting, scientists reported connections between C9ORF72 loss and gain of function. Their talks brimmed with new biology implicating autophagy, the cellular stress response, and RAN translation off introns.
Swine that express mutated human SOD1 exhibit the hallmarks of ALS pathology, including aggregates and degeneration in motor neurons, preceded by a lengthy preclinical phase.
In mice, a daily low dose restored regulatory T cells and held off symptoms of a demyelinating disease. Could an old, over-the-counter drug help with ALS, in which similar cells decline?
The Food and Drug Administration has said it will accept cognition alone as the basis for approval in preclinical AD. Now what? Industry is confronting the challenge to show robust and meaningful change.
In a new take on mosaicism and Alzheimer’s, scientists claim that APP mRNAs convert into DNA and reinsert into the genome. Full of mutations, these “genomic cDNAs” crop up in aging and sporadic AD.
In Barcelona, data ran the gamut from a few hopeful little hints on new treatments to mixed signals on familiar players, and failed drugs thrown on the scrap heap.