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US 2025/0019697 A1Published application
Modified u6 promoter system for tissue specific expression
AssigneeResearch Institute At Nationwide Children's Hospital
InventorScott Quenton Harper
Life of this patent
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Abstract
The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.
Claims21
A nucleic acid molecule comprising a modified U6 promoter sequence, mature guide strand of a miRNA comprising at least one detargeting sequence and 5-6 thymidines at the 5′ end.
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Dependencies are inferred from the claim text.