Anti-GA therapy based on AAV

Abstract

The aspects of the description relate to compositions and methods for the diagnosis and / or treatment of certain neurodegenerative diseases, for example, diseases related to non-ATG repeat-associated translation proteins (RANs), such as amyotrophic lateral sclerosis (ALS); in some modalities, the description relates to recombinant adeno-associated viruses (rAAVs) expressing antibodies and antigen-binding fragments thereof that bind to poly(GA) RAN proteins; in some modalities, the description relates to methods for treating a RAN-related disease by administering rAAVs to a subject in need.