@Rothstein_Lab
link.springer.com TDP-43-related cryptic splicing reflects pathological heterogeneity in amyotrophic lateral sclerosis Acta Neuropathologica Communications - Amyotrophic lateral sclerosis (ALS) involves TAR DNA-binding protein 43 (TDP-43) dysfunction, leading to cryptic splicing, including UNC13A cryptic exon... 
pubs.acs.org Selective Brain-Penetrant TTBK1 Inhibitors Modulate TDP-43 Pathology and Rescue Cognitive Deficits... Abstract. Transactive response DNA-binding protein of 43 kDa (TDP-43) is a pathological hallmark of neurodegenerative disorders, including amyotrophic late 
rupress.org EPHA2/CD44-directed trafficking enhances endosomal leakiness and antisense therapy delivery A scavenger receptor (CD44) and a receptor tyrosine kinase (EPHA2) join forces to mediate internalization of therapeutic ASOs into endosomes, which are the 
www.biorxiv.org Astrocyte dysfunction distinguishes monozygotic twin C9orf72 expansion carriers discordant for... Hexanucleotide repeat expansions in C9orf72 are the most common genetic cause of amyotrophic lateral sclerosis, yet many carriers remain asymptomatic for decades or never develop disease. This... 