# US 2014/0193856 A1 · Control of gene expression

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- Number: US 2014/0193856 A1
- Document ID: US20140193856A1
- Status: Published application
- Kind: A1
- Application number: 14/137,737
- Filed: 2013-12-20
- Published: 2014-07-10
- Earliest claimed: 1998-03-20
- Assignees: Commonwealth Scientific and Industrial Research Organisation
- Inventors: Michael Wayne Graham; Robert Norman Rice; Peter Michael Waterhouse
- Web page: https://patentgrep.com/patent/US20140193856A1

## CPC

- C12N 15/113: Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)}
- A61K 48/00: Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61P 31/04: Antibacterial agents
- A61P 31/12: Antivirals
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C12N 9/1051: {Hexosyltransferases (2.4.1)}
- C12N 9/127: {RNA-directed RNA polymerase (2.7.7.48), i.e. RNA replicase}
- C12N 9/503: {derived from viruses}
- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- C12N 15/1131: {against viruses}
- C12N 15/63: Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N 15/69: Increasing the copy number of the vector
- C12N 15/8216: {Methods for controlling, regulating or enhancing expression of transgenes in plant cells}
- C12N 15/8218: {Antisense, co-suppression, viral induced gene silencing [VIGS], post-transcriptional induced gene silencing [PTGS]}
- C12N 15/8283: {for virus resistance}
- C12N 15/85: for animal cells
- A01K 2217/05: Animals comprising random inserted nucleic acids (transgenic)
- C12N 2310/111: spanning the whole gene, or a large part of it
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/531: Stem-loop; Hairpin
- C12N 2320/10: in screening processes
- C12N 2320/30: Special therapeutic applications
- C12N 2330/30: chemically synthesised
- C12N 2330/50: Biochemical production, i.e. in a transformed host cell
- C12N 2330/51: Specially adapted vectors
- C12N 2800/108: episomal vectors
- C12N 2830/00: Vector systems having a special element relevant for transcription
- C12N 2830/002: inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
- C12N 2830/15: chimeric enhancer/promoter combination
- C12N 2830/38: being a stuffer
- C12N 2830/42: being an intron or intervening sequence for splicing and/or stability of RNA
- C12N 2830/55: from bacteria
- C12N 2830/60: from viruses
- C12N 2840/20: translation of more than one cistron
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- 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)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A01K: ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS

## Abstract

The present invention relates generally to a method of modifying gene expression and to synthetic genes for modifying endogenous gene expression in a cell, tissue or organ of a transgenic organism, in particular a transgenic animal or plant. More particularly, the present invention utilizes recombinant DNA technology to post-transcriptionally modify or modulate the expression of a target gene in a cell, tissue organ or whole organism, thereby producing novel phenotypes. Novel synthetic genes and genetic constructs which are capable of repressing delaying or otherwise reducing the expression of an endogenous gene or target gene in an organism when introduced thereto are also provided.

## Claims

### Claim at position 1

1-43. (canceled)

### Claim 44

A double-stranded DNA construct which is capable of delaying, repressing, or otherwise reducing the expression of a target gene, wherein the double-stranded DNA construct comprises a foreign nucleic acid molecule comprising multiple copies of a nucleotide sequence that is more than 20 nucleotides long and which is substantially identical to a region of the target gene, and wherein the foreign nucleic acid molecule is placed operably under the control of both a first promoter sequence and a second promoter sequence which are each operable in a cell, wherein said promoters are located at the distal and proximal ends of the foreign nucleic acid molecule.

### Claim 45 (depends on 44)

The double-stranded DNA construct of claim 44, wherein the multiple copies are arranged so as to form an inverted repeat sequence between the first promoter sequence and the second promoter sequence.

### Claim 46 (depends on 44)

The double-stranded DNA construct of claim 44, wherein the multiple copies are arranged so as to form an interrupted palindrome sequence between the first promoter sequence and the second promoter sequence.

### Claim 47 (depends on 44)

The double-stranded DNA construct of claim 44, wherein the target gene is in an animal cell.

### Claim 48 (depends on 47)

The double-stranded DNA construct of claim 47, wherein the animal cell is a human cell.

### Claim 49 (depends on 47)

The double-stranded DNA construct of claim 47, wherein the target gene is a gene that is contained within the genome of the animal cell.

### Claim 50 (depends on 49)

The double-stranded DNA construct of claim 49, wherein the animal cell is an invertebrate animal cell.

### Claim 51 (depends on 50)

The double-stranded DNA construct of claim 50, wherein the invertebrate animal cell is an insect cell.

### Claim 52 (depends on 47)

The double-stranded DNA construct of claim 47, wherein the target gene is a gene that is contained within the genome of a pathogen of the animal cell.

### Claim 53 (depends on 44)

The double-stranded DNA construct of claim 44, wherein the nucleotide sequence of the first promoter sequence is different from nucleotide sequence of the second promoter sequence.

### Claim 54 (depends on 44)

The double-stranded DNA construct of claim 44, wherein the first promoter sequence and the second promoter sequence are each operable in a eukaryotic cell.

### Claim 55 (depends on 44)

The double-stranded DNA construct of claim 44, wherein the first promoter sequence and the second promoter sequence are each operable in a prokaryotic cell.

### Claim 56 (depends on 44)

A cell comprising the double-stranded DNA construct of claim 44.

### Claim 57 (depends on 12)

A prokaryotic cell comprising the double-stranded DNA construct of claim 12.

### Claim 58 (depends on 57)

The prokaryotic cell of claim 57, wherein the target gene is a non-endogenous gene to the prokaryotic cell.

### Claim 59 (depends on 56)

The prokaryotic cell of claim 56, wherein the non-endogenous gene is a gene that is contained within the genome of an insect cell.

### Claim 60 (depends on 44)

A process for producing a nucleic acid molecule comprising culturing a cell transfected with the double-stranded DNA construct of claim 44 under conditions such that the cell produces the nucleic acid molecule.

### Claim 61 (depends on 60)

The process of claim 60, wherein the cell is a prokaryotic cell.