Patent ID: 8524499
Filing Date: 2013-09-03
Classification: A01K,C12N

Abstract:
1. A method of expressing a transcript of interest specifically in motor neurons of a subject, said method comprising: providing a viral vector comprising a nucleic acid construct comprising: an enhancer which functions in human brain motor neurons or spinal cord motor neurons, wherein the enhancer comprises a nucleotide sequence of SEQ ID NO: 5; a basal promoter; a nucleic acid encoding the transcript of interest; and a 3′ control region, wherein the enhancer, the basal promoter, the nucleic acid encoding the transcript of interest, and the 3′ control region are operably linked for expression of the transcript of interest and administering the viral vector directly to the subject's spinal cord or brain that comprise the motor neurons, or to a muscle innervated by the motor neurons wherein the vector is delivered to the motor neurons of the subject and the transcript of interest is expressed therein.