# US 12,109,224 B2 · USB1 directed regulation of miRNAs related hematopoietic differentiation and affected in multiple forms of leukemia

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- Number: US 12,109,224 B2
- Document ID: US12109224B2
- Status: Granted
- Kind: B2
- Application number: 17/466,448
- Filed: 2021-09-03
- Published: 2022-03-24
- Granted: 2024-10-08
- Earliest claimed: 2020-09-04
- Assignees: The Regents of the University of Colorado, a body corporate; Washington University
- Applicants: The Regents of the University of Colorado, a body corporate; Washington University
- Inventors: Roy R. Parker; Siddharth Shukla; Luis F. Z. Batista; Hochang Jeong
- Web page: https://patentgrep.com/patent/US12109224B2

## CPC

- A61K 31/713: Double-stranded nucleic acids or oligonucleotides
- A61K 31/4375: the heterocyclic ring system containing a six-membered ring having nitrogen as a ring heteroatom, e.g. quinolizines, naphthyridines, berberine, vincamine
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)

## Abstract

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.

## Claims

### Claim 1

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: miR-142-5p, miR-199a-3p, and miR-223-3p.

### Claim 2 (depends on 1)

The method of claim 1, wherein the cell comprises a human cell.