Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration

摘要

The present application provides materials and methods for treating a patient with amyotrophic lateral sclerosis (ALS) and / or frontotemporal lobular degeneration (ftld), both ex vivo and in vivo. In addition, the present application provides materials and methods for editing to modulate c9orf72 gene expression, function or activity in a cell by genome editing.