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.
Type:
Application
Filed:
June 15, 2017
Publication date:
October 31, 2019
Applicant:
CRISPR Therapeuticas AG
Inventors:
Ante Sven LUNDBERG, Samarth KULKARNI, Lawrence KLEIN, Hari Kumar PADMANABHAN