abstract |
The present application provides materials and methods for treating a patient with Amyotrophic Lateral Sclerosis (ALS) and/or Frontaltemporal Lobular Degeneration (FTLD), both ex vivo and in vivo . In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the C9ORF72 gene in a cell by genome editing. |