# US 2013/0156748 A1 · Use of a hspc117 molecule as rna ligase

> Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt

- Number: US 2013/0156748 A1
- Document ID: US20130156748A1
- Status: Published application
- Kind: A1
- Application number: 13/814,854
- Filed: 2011-08-30
- Published: 2013-06-20
- Earliest claimed: 2010-08-30
- Assignees: Imba—Institut FüR Molekulare Biotechnologie GmbH
- Inventors: Johannes Popow; Stefan Weitzer; Javier Martinez; Karl Mechtler; Alexander Schleiffer
- Web page: https://patentgrep.com/patent/US20130156748A1

## CPC

- C12N 9/93: {Ligases (6)}
- A61P 25/00: Drugs for disorders of the nervous system
- A61P 31/12: Antivirals
- A61P 35/00: Antineoplastic agents
- C12Q 1/68: involving nucleic acids
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES

## Abstract

The present invention relates to the use of HSPC117 molecules as RNA ligase, methods of ligating RNA molecules, kits for these methods and uses and transgenic cells.

## Claims

### Claim 1

The use of a HSPC117 molecule as RNA ligase, preferably for the transfer of a first RNA end to a second RNA end, in particular preferred wherein the first end comprises a 3′ phosphate, preferably a 2′,3′-cyclic phosphate, and/or wherein the second end comprises a 5′-OH terminus.

### Claim 2 (depends on 1)

The use of claim 1 for inter- or intra-strand ligation.

### Claim 3 (depends on 1)

The use of claim 1 wherein the RNA is double-stranded.

### Claim 4 (depends on 1)

The use of claim 1 for RNA splicing.

### Claim 5 (depends on 1)

The use of claim 1 wherein the RNA is a mRNA or tRNA.

### Claim 6 (depends on 1)

The use of claim 1 wherein the RNA is 2 to 3000 nucleotides or basepairs in length.

### Claim 7 (depends on 1)

The use of claim 1 wherein the HSPC117 molecule is of any one of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 5, SEQ ID NO: 6, SEQ ID NO: 7, SEQ ID NO: 8, SEQ ID NO: 9, SEQ ID NO: 10, SEQ ID NO: 11, SEQ ID NO: 13, SEQ ID NO: 15, SEQ ID NO: 17, SEQ ID NO: 19, SEQ ID NO: 21, SEQ ID NO: 23.

### Claim 8 (depends on 1)

The method of ligating at least two RNA molecules comprising using a HSPC117 molecule according to claim 1, preferably comprising contacting the at least two RNA molecules with said HSPC117 molecule in a cell, or preferably comprising contacting the at least two RNA molecules with said HSPC117 molecule in vitro, in particular preferred further comprising providing the HSPC117 molecule in a complex, preferably with spliceosomal particles, preferably wherein the complex comprises a DEAD box helicase DDX1 molecule, a FAM98B molecule, a CGI-99 molecule, ASW or any combination thereof.

### Claim 9

A kit suitable for performing RNA ligation, comprising
a HSPC117 molecule,
a RNA ligase reaction buffer comprising buffer components and one or more metal ions selected from Mg2+, Mn2+, Ni2+ or mixtures
thereof, for use in a final concentration range of ca 0.1-20 mM, preferably 1-10 mM, in particular preferred 2-5 mM, optionally further comprising a RNA molecule with 2′,3′-cyclic phosphate, preferably further with a label.

### Claim 10

A transgenic cell comprising an exogenously expressed HSPC117 molecule, preferably with an inducible promoter.

### Claim 11

A HSPC117 knock-out cell or cell with reduced inhibited endogenous HSPC117 expression.

### Claim 12 (depends on 10)

Cell according to claim 10 being a mammal cell.

### Claim 13

A method of reducing RNA ligase activity, in particular RNA>p ligase activity, in a cell comprising inhibiting a HSPC117 molecule in a cell, preferably by knock-out or RNAi.

### Claim 14 (depends on 13)

The method of claim 13, characterized in that inhibition is by reducing expression of an HSPC117 molecule, preferably an endogenous HSPC117 molecule, in said cell.

### Claim 15 (depends on 13)

The method of claim 13 for reducing tRNA production or processing in said cell.

### Claim 16 (depends on 10)

The use of a cell according to claim 10 for RNA ligation or splicing studies, preferably to modulate tiRNA amounts in a cell.

### Claim 17

A method of reducing tumor cell growth or a disease or infection dependent on host polymerases, such as hepatitis delta virus infection, comprising administering an HSPC117 molecule inhibitor to said cell, or of treating a disease in a subject associated with dysfunctional tRNA splicing, preferably pontocerebellar hypoplasia, comprising administering a HSPC117 molecule to said subject.