Induced Pluripotent Stem Cells to Research Proposal

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3.3 Data Collection

Is maternal UBE3A active following iPS treatment: Data will be gathered on the iPS-treated mice via positron emission tomography, and in vivo brain slice preparation, and Western Blot Analysis. H1 will essentially be ascertained following these tests.

Does iPS treatment rescue the motor and cognitive deficits associated with Angelman Syndrome: Data will be gathered from testing the treated mice in scientifically recognized tests of cognitive ability in a mouse model. This project proposes using the water maze test, the electric shock test, and the submerged platform test. H2 will effectively be answered using the data gleaned from these tests.

4. Conclusion

4.1. Potential Therapeutic and Other Considerations

The potential of using iPS treatment to rescue/alleviate the severe motor and cognitive deficits witnessed in Angelman Syndrome is theoretically viable. Reliable mouse models of AS exist with which to run the tests. The technology needed to tease iPS stem cells into fully functioning brain cells that express the UBE3A gene product is available and viable to conduct in a controlled setting.

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Therefore, the proposal as set forth is a logical one which addresses an urgent need in an uncharted territory -- treating imprinted neurological disorders using iPS treatment. If successful, the potential to treat not only other imprinted neurological disorders (Rett Syndrome, Prader Willi Syndrome) but to also treat other neurological disorders that share similar dysfunctional brain pathway etiologies, is immense. If the hypotheses in the proposal are proven, the door to researching viable and cutting-edge treatments for neurological disorders harkens to the researchers. Devastating disorders that cost immeasurable amounts of human suffering and untold millions of dollars in health care and long-term care, could be cured. A novel therapeutic that bypasses the ethical, political, and social backlash associated with stem cell research, could potentially change the course of mankind......

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