Loading patent…
AssigneesThe Regents of the University of Colorado, a body corporate, Washington University
InventorsRoy R. Parker, Siddharth Shukla, Luis F.Z. Batista, Hochang Jeong
Estimating the term…
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 the cell of a subject in need thereof, comprising the step of administering a therapeutically effective amount of a USB1 inhibitor that inhibits the activity of USB1, or downregulates of the expression of usb17.
A method of upregulating miRNA expression in the cell of a subject in need thereof, comprising the step of administering a therapeutically effective amount of a PAPD5/7 inhibitor that inhibits the activity of PAPD5/7, or downregulates of the expression of papd5/7, wherein said cell is a human cell having one or more homozygous or compound heterozygous mutations in the usb1 gene
A method of modulating miRNA expression, comprising the step of contacting a therapeutically effective amount of a USB1 inhibitor, or a PAPD5/7 inhibitor with cell.
Dependencies are inferred from the claim text.