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.