| abstract |
The present invention provides compositions and methods for treating neurological disorders, such as in particular amyotrophic lateral sclerosis, frontotemporal degeneration and Alzheimer's disease. In order to treat the underlying etiology of the disease and/or alleviate one or more symptoms of the disease using the compositions and methods described herein, there is a neurological disorder, such as a neurological disorder associated with TAR-DNA binding protein (TDP)-43 aggregation. Patients may be administered an inhibitor of cytochrome P450 (CYP450) isoform protein 51A1 (CYP51A1), also referred to herein as lanosterol 14-alpha demethylase. The inhibitor of CYP51A1 may be a small molecule, an anti-CYP51A1 antibody or antigen binding fragment or compound thereof, such as an interfering RNA molecule that attenuates CYP51A1 expression. Patients that can be treated using the compositions and methods described herein include patients expressing a mutant TDP-43 isotype protein containing mutations associated with TDP-43-promoting aggregation and toxicity. |