# US 9,040,491 B2 · Compositions, methods and kits based on small nuclear RNAs

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- Number: US 9,040,491 B2
- Document ID: US9040491B2
- Status: Granted
- Kind: B2
- Application number: 11/897,052
- Filed: 2007-08-29
- Published: 2008-08-14
- Granted: 2015-05-26
- Earliest claimed: 2006-08-30
- Assignees: The Trustees of the University of Pennsylvania
- Applicants: Gideon Dreyfuss; Jeongsik Yong; Tracey (Golembe) Polsky
- Inventors: Gideon Dreyfuss; Jeongsik Yong; Tracey (Golembe) Polsky
- Web page: https://patentgrep.com/patent/US9040491B2

## CPC

- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- C12N 15/11: DNA or RNA fragments; Modified forms thereof (DNA or RNA not used in recombinant technology, C07H21/00); {Non-coding nucleic acids having a biological activity}
- C12N 2310/3519: Fusion with another nucleic acid
- C12N 2320/32: Special delivery means, e.g. tissue-specific
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

The present invention includes methods for modulating the expression of a gene or mRNA in a cell using small nuclear RNAs comprising specific sequence and structural features and further comprising a nucleic acid cargo.

## Claims

### Claim 1

A composition comprising a nucleic acid cargo sequence and an isolated snRNA, wherein said cargo sequence is linked to said snRNA, further wherein said snRNA consists of:
(a) an Sm site, wherein said Sm site is selected from the group consisting of SEQ ID NO. 1, SEQ ID NO. 2, and SEQ ID NO. 3;
(b) a stem-loop structure positioned 3′ to said Sm site, wherein the number of nucleotides between the Sm site and the 3′ stem loop is between 1 and 5 nucleotides, further wherein said stem-loop structure comprises from about 7 to about 12 base pairs in the stem and from about 4 to about 17 nucleotides in the loop; and
(c) nucleotides located 3′ from the stem-loop structure consisting of 5-10 nucleotides.

### Claim 2 (depends on 1)

The composition of claim 1, wherein said cargo sequence is an antisense oligonucleotide.

### Claim 3 (depends on 1)

The composition of claim 1, wherein said cargo sequence is a small interfering RNA (siRNA).

### Claim 4 (depends on 1)

The composition of claim 1, wherein said cargo sequence is a splice-switching oligonucleotide.

### Claim 5 (depends on 1)

The composition of claim 1, wherein said cargo sequence is an RNA binding protein decoy.

### Claim 6 (depends on 1)

A pharmaceutical composition comprising the isolated snRNA of claim 1.

### Claim 7

A kit for modulating the expression of a gene in a cell, said kit comprising a composition comprising a nucleic acid cargo sequence and an isolated snRNA, wherein said cargo sequence is linked to said snRNA, further wherein said snRNA consists of:
(a) an Sm site, wherein said Sm site is selected from the group consisting of SEQ ID NO. 1, SEQ ID NO. 2, and SEQ ID NO. 3;
(b) a stem-loop structure positioned 3′ to said Sm site, wherein the number of nucleotides between the Sm site and the 3′ stem loop is between 1 and 5 nucleotides, further wherein said stem-loop structure comprises from about 7 to about 12 base pairs in the stem and from about 4 to about 17 nucleotides in the loop; and
(c) nucleotides located 3′ from the stem-loop structure consisting of 5-10 nucleotides;
wherein, said kit further comprising an applicator and an instructional material for the use thereof.

### Claim 8

An isolated molecule consisting of a nucleic acid cargo sequence and an isolated snRNA, wherein said snRNA:
(a) an Sm site, wherein said Sm site is selected from the group consisting of SEQ ID NO. 1, SEQ ID NO. 2, and SEQ ID NO. 3;
(b) a stem-loop structure positioned 3′ to said Sm site, wherein the number of nucleotides between the Sm site and the 3′ stem loop is between 1 and 5 nucleotides, further wherein said stem-loop structure comprises from about 7 to about 12 base pairs in the stem and from about 4 to about 17 nucleotides in the loop; and
(c) nucleotides located 3′ from the stem-loop structure consisting of 5-10 nucleotides.