# US 2018/0023142 A1 · Micrornas in neurodegenerative disorders

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- Number: US 2018/0023142 A1
- Document ID: US20180023142A1
- Status: Published application
- Kind: A1
- Application number: 15/699,546
- Filed: 2017-09-08
- Published: 2018-01-25
- Earliest claimed: 2011-10-11
- Assignees: The Brigham and Women S Hospital, Inc.; The General Hospital Corporation
- Applicants: The Brigham and Women`s Hospital, Inc.; The General Hospital Corporation
- Inventors: Howard Weiner; Oleg Butovsky; Merit Cudkowicz; James Berry
- Web page: https://patentgrep.com/patent/US20180023142A1

## CPC

- C12Q 1/6883: for diseases caused by alterations of genetic material
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 21/02: Muscle relaxants, e.g. for tetanus or cramps
- A61P 25/00: Drugs for disorders of the nervous system
- 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)}
- C12N 2310/113: targeting other non-coding nucleic acids, e.g. antagomirs
- C12N 2310/3231: having an additional ring, e.g. LNA, ENA
- C12Q 2600/158: Expression markers
- C12Q 2600/178: miRNA, siRNA or ncRNA
- 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
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

Provided herein are methods of treating a neurodegenerative disorder such as Amyotrophic Lateral Sclerosis (ALS) or Multiple Sclerosis (MS) that include administering to a subject at least one inhibitory nucleic acid that decreases the level or activity of microRNA has-miR-155.

## Claims

### Claim 1

A method of treating a subject having Alzheimer's Disease (AD), the method comprising identifying a subject who has AD and administering to the subject having AD at least one inhibitory nucleic acid comprising a sequence that is complementary to mature microRNA hsa-miR-155 having the sequence of SEQ ID NO:58, wherein the inhibitory nucleic acid reduces a level or activity of hsa-miR-155 in monocytes of the subject.

### Claim 2 (depends on 1)

The method of claim 1, wherein the at least one inhibitory nucleic acid is an antisense oligonucleotide.

### Claim 3 (depends on 1)

The method of claim 1, wherein the inhibitory nucleic acid comprises a sequence that is complementary to at least five nucleotides of the seed sequence of mature microRNA hsa-miR-155

### Claim 4 (depends on 1)

The method of claim 1, wherein the at least one inhibitory nucleic acid is administered intravenously or intrathecally.

### Claim 5 (depends on 1)

The method of claim 1, wherein the at least one inhibitory nucleic acid is administered by injection into the cerebrospinal fluid.

### Claim 6 (depends on 1)

The method of claim 1, wherein the at least one inhibitory nucleic acid is complexed with one or more cationic polymers or cationic lipids.

### Claim 7 (depends on 1)

The method of claim 1, wherein the at least one inhibitory nucleic acid is complementary to a contiguous sequence of at least 12 nucleotides present in hsa-miR-155.

### Claim 8 (depends on 1)

The method of claim 1, wherein the at least one inhibitory nucleic acid is complementary to a contiguous sequence of at least 14 nucleotides present in hsa-miR-155.

### Claim 9 (depends on 1)

The method of claim 1, wherein the at least one inhibitory nucleic acid is complementary to a contiguous sequence of at least 15 nucleotides present in hsa-miR-155.

### Claim 10 (depends on 1)

The method of claim 1, wherein the at least one inhibitory nucleic acid comprises at least one locked nucleic acid (LNA).

### Claim 11 (depends on 1)

The method of claim 1, wherein the subject who has AD is human.