Citations 1
Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion
O’Rourke, Jacqueline G., Meera, Pratap , Muhammad, A.K.M. Ghulam, Grant, Sharday, Simpkinson, Megan, Bell, Shaughn , Carmona, Sharon, Ornelas, Loren, Sahabian, Anais , Petrucelli, Leonard , Baughn, Michael W. , Ravits, John , Harms, Matthew B. , Rigo, Frank , Bennett, C. Frank , Otis, Thomas S. , Svendsen, Clive N. , Baloh, Robert H. , Sareen, Dhruv , Gendron, Tania F.
Science Translational Medicine | October 23, 2013 | Volume: 5 | Issue: 208 | Pages: No page available
Antisense oligonucleotides can correct disease-specific phenotypes in cultured motor neurons differentiated from iPSCs derived from ALS patients with a C9ORF72 repeat expansion.