METHODS TO TREAT NEUROLOGICAL DISEASES

摘要

Disclosed is a method of treating a subject who has a neurological disease. The neurological disease may be associated with altered C9ORF72 protein activity. In one aspect, the method includes a step of administering an effective dose of a pharmaceutical composition to a subject in need thereof, thereby rescuing the defects associated with altered C9ORF72 protein activity. Also described are methods for identifying a compound for inhibiting motor neuron degeneration.