Ching-Hwa Sung, The Betty Neuwirth Lee and Chilly Professor in Stem Cell Research
Invention: Isolated promoter region for Tctex-1 gene, which can be used to mark adult neural stem cells
Potential commercial application: Therapeutics for CNS injury, stroke, and depression, and hypothalamic control of obesity
TcTex-1 is expressed specifically in progenitor cells in the hippocampus, the hypothalamus and the cortex of the adult brain. Marker genes, such as GFP, can be placed under the control of the Tctex-1 promoter in order to mark adult neural stem cells. This can be useful for targeting gene therapies to neural stem cells, and in drug screens, to identify small molecules that can induce a patients own neural stem cells to profilerate and differereniate. On a parallel track, cancer research has recently come to focus on identifying and killing cancer stem cells that appear to be crucial in metastases and resistance to treatment.
The Sung lab at Weill Cornell Medicine has used a Tctex-1-promoter construct to selectively label neural progenitors in these regions. In a similar fashion, this invention allows introduction of any genetic sequence selectively into neural progenitors including siRNA, microRNA, or a sequence encoding any protein of interest.
Cornell's patent application covers the Tctex-1 regulatory sequence in mouse and human, and methods to use these sequences for assays, isolation of progenitor populations, manipulation of cells in vitro, and gene therapy.
The invention faciliates research, development and delivery of CNS stem cell therapies, as well as R&D in modulators of endogenous neurogenesis. As Txtex-1 marks progenitors in the hypothalamus, it may also provide a tool to modulate energy balance and to treat obesity (see for example, Kokoeva MV (2005) Neurogenesis in the hypothalamus of adult mice: potential role in energy balance. Science. 310(5748):679-83.)
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