Associated Papers
CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro.
Meijboom KE et al.
Nature Communications, 2022
G-quadruplex-binding small molecules ameliorate C9orf72 FTD/ALS pathology in vitro and in vivo.
Simone R et al.
Embo Molecular Medicine, 2018
Screening for novel hexanucleotide repeat expansions at ALS- and FTD-associated loci.
He F et al.
Neurology. Genetics, 2016
G-quadruplex structures formed by expanded hexanucleotide repeat RNA and DNA from the neurodegenerative disease-linked C9orf72 gene efficiently sequester and activate heme.
Grigg JC et al.
Plos One, 2014
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