Cornell Research Foundation Inc.

Takahiro Nakano

Steven A Goldman

Neeta Singh Roy

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.

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

The present invention is directed to a method of isolating an enriched or purified population of motor neurons from a population of embryonic stem cells. This method involves providing a population of embryonic stem cells and selecting a promoter or enhancer which functions only in the motor neurons selected. A nucleic acid molecule encoding a marker protein under control of the promoter or enhancer is introduced into the induced population of embryonic stem cells. The motor neurons are allowed to express the marker protein and, the cells expressed in the marker protein are separated from the population of embryonic stem cells. The population of embryonic stem cells can be induced to produce a mixed population of cells comprising motor neurons before or after a nucleic acid molecule encoding the marker protein under control of the promoter enhancer is introduced into the population of embryonic stem cells. As a result, an enriched or purified population of motor neurons is isolated.

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New York, NY, United States of America

Takahiro Nakano