US 8,524,499 B2 · Regulatory sequences that direct gene expression to spinal motor neurons and uses thereof

Abstract

The present invention relates to an enhancer which functions only in human brain and/or spinal cord motor neurons, where the enhancer comprises a nucleotide sequence of SEQ ID NO: 5, SEQ ID NO: 10, or SEQ ID NO: 16. The enhancer can be utilized as part of a nucleic acid construct which also has a nucleic acid encoding a marker protein or a therapeutic protein, a 3′ control region, and, optionally, a basal promoter, where these components are positioned with respect to one another to permit expression of the marker protein or the therapeutic protein. The enhancer of the present invention is useful in a method of isolating an enriched or purified population of motor neurons from a mixed population of human brain and/or spinal cells. In addition, the enhancer of the present invention can be used in a method of therapeutically targeting motor neurons.

Claims26

Dependent claims

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.

5 more elements

Dependencies are inferred from the claim text.

Regulatory sequences that direct gene expression to spinal motor neurons and uses thereof

Life of this patent

Regulatory sequences that direct gene expression to spinal motor neurons and uses thereof

Life of this patent

Abstract

Claims26