New XCI Inhibitors as Potential Rett Syndrome Therapeutics
- 详细技术说明
- This invention provides methods of treating a subject having a dominant X-linked disease, the method comprising administering to the subject an X chromosome inactivation factor (XCIF) inhibitor in an
- *Abstract
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This invention provides methods of treating a subject having a dominant X-linked disease, the method comprising administering to the subject an X chromosome inactivation factor (XCIF) inhibitor in an amount effective for inducing expression a target X-linked gene. The invention provides small molecule and oligonucleotide XCIF inhibitors. In some embodiments, the X-linked gene is MECP2 and the X-linked disease is Rett Syndrome.
- *IP Issue Date
- None
- *IP Type
- Other Patent
- 国家
- Not Available
- 申请号码
- None
- 国家/地区
- 美国
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