Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo
Gabriela Toro Cabrera, Katharina E. Meijboom, Abbas Abdallah, Hélène Tran, Zachariah W. Foster, Alexandra Weiss, Nicholas Wightman, Rachel Stock, Tania F. Gendron, Alisha M. Gruntman, Anthony Giampetruzzi, Leonard Petrucelli, Robert H. Brown, Christian Mueller
Topics & Concepts
C9orf72Amyotrophic lateral sclerosisBiologyFrontotemporal dementiaTransgeneTrinucleotide repeat expansionmicroRNAGeneCell biologyGeneticsPathologyMedicineDiseaseDementiaAlleleAmyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders ResearchRNA Research and Splicing