CRISPR Therapeutics AG
Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders

Abstract:

The present disclosure provides materials and methods for treating a patient with one or more conditions associated with SOD1 whether ex vivo or in vivo. In addition, the present disclosure provides materials and methods for editing and/or modulating the expression of SOD1 gene in a cell by genome editing.