MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND/OR FRONTAL TEMPORAL LOBULAR DEGENERATION.

摘要

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.