Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion

• DOI: 10.1126/scitranslmed.3007529
• Publication Date: 2013-10-23T18:25:43Z
• Authors: Dhruv Sareen; Jacqueline G. O’R...; Pratap Meera; A. K. M. G. Muhammad; Sharday Grant; Megan Simpkinson; Shaughn Bell; Sharon Carmona; Loren Ornelas; Anais Sahabian; Tania Gendron; Leonard Petrucelli; Michael Baughn; John Ravits; Matthew B. Harms; Frank Rigo; C. Frank Bennett; Thomas S. Otis; Clive N. Svendsen; Robert H. Baloh
• Keywords: C9ORF72

Antisense oligonucleotides can correct disease-specific phenotypes in cultured motor neurons differentiated from iPSCs derived ALS patients with a C9ORF72 repeat expansion.