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AssigneesThe Regents of the University of Colorado, a body corporate, Washington University
InventorsRoy R. Parker, Siddharth Shukla, Luis F. Z. Batista, Hochang Jeong
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The invention includes methods and compositions for the treatment and prevention of a disease or condition resulting from dysregulation of USB1 and resulting expression of downstream miRNAs. In one preferred embodiment, the invention may include inhibiting the expression or activity of USB1 resulting in the downregulation of downstream miRNA expression. In alternative embodiments, the invention includes methods and compositions for the treatment and prevention of a disease or condition resulting from dysregulation of miRNAs downstream of USB1, namely through the inhibition of PAPD5 and/or PAPD7, resulting in the upregulation of said downstream miRNA expression.
A method of downregulating miRNA expression in a cell comprising the step of downregulating the expression of usb17 in the cell via introduction of a CRISPR-mediated gene therapy that knocks out usb17 in the cell, and wherein the downregulated miRNA include at least one of the following:
Dependencies are inferred from the claim text.