Mohammad M Doroudchi

US Patent:

20080181872, Jul 31, 2008

Filed:

Jan 7, 2008

Appl. No.:

11/970138

Inventors:

Mohammad Doroudchi - San Ramon CA, US

International Classification:

A61K 48/00
C12N 15/00
C12Q 1/70
A61P 25/00
A61P 19/00
A61P 3/00
A61P 27/02

US Classification:

424 932, 4353201, 435 5

Abstract:

The invention relates to adeno-associated virus (AAV) vectors encoding superoxide dismutase (SOD), where the AAV vector encoding SOD (AAV-SOD) may be used to deliver the SOD gene to target cells. The target cells may be within a subject having a disease or condition for which delivery of SOD to the target cells provides a therapeutic benefit and/or a therapeutic effect on the subject. In another aspect, the invention relates to a model system for screening compounds for efficacy in treatment of amyotrophic lateral sclerosis (ALS). The model system may comprise a plurality of cells transduced with an AAV vector encoding an SOD gene; the transduced cells may exhibit a phenotypic change associated with ALS. The model system of the invention may be used to screen compounds for efficacy in treatment of ALS using