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COMPOSITIONS AND METHODS FOR DIAGNOSING AND TREATING NUCLEOTIDE REPEAT DISORDERS
专利权人:
University of Virginia Patent Foundation
发明人:
Erin P. FOFF,Benjamin W. PUROW
申请号:
US15156626
公开号:
US20160340732A1
申请日:
2016.05.17
申请国别(地区):
US
年份:
2016
代理人:
摘要:
The present application discloses compositions and methods useful for diagnosing and treating nucleotide repeat disorders. An in silico analysis indicates a striking and specific miR-NRD homology. Validated miR target prediction software was used to assess each NRD nucleotide repeat expansion for potential miR homology. The striking degree of matched homology and the one-to-one expansion-to-miR ratio strongly support their relevance to the corresponding NRD and serve as a basis for therapeutic treatments. It is also disclosed herein that NETO is involved in mediating C9-repeat glutamate excitotoxicity, thus representing a novel therapeutic target.
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