# US 10,870,853 B2 · Compositions and methods for modulating neuronal excitability and motor behavior

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- Number: US 10,870,853 B2
- Document ID: US10870853B2
- Status: Granted
- Kind: B2
- Application number: 16/148,015
- Filed: 2018-10-01
- Published: 2019-05-16
- Granted: 2020-12-22
- Earliest claimed: 2013-10-28
- Assignees: Icahn School of Medicine at Mount Sinai; The Rockefeller University
- Applicants: Icahn School of Medicine At Mount Sinai; The Rockefeller University
- Inventors: Anne Schaefer; Paul Greengard
- Web page: https://patentgrep.com/patent/US10870853B2

## 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)}
- A01K 67/0275: Genetically modified vertebrates, e.g. transgenic
- A01K 67/0276: Knock-out vertebrates
- A61K 38/185: {Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3}
- A61K 49/0008: {Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure}
- G01N 33/5058: {Neurological cells}
- A01K 2217/052: inducing gain of function
- A01K 2217/075: inducing loss of function, i.e. knock out
- A01K 2217/203: Animal model comprising inducible/conditional expression system, e.g. hormones, tet
- A01K 2227/105: Murine
- A01K 2267/0356: Animal model for processes and diseases of the central nervous system, e.g. stress, learning, schizophrenia, pain, epilepsy
- A61P 25/00: Drugs for disorders of the nervous system
- A61P 25/08: Antiepileptics; Anticonvulsants
- C12N 2310/141: MicroRNAs, miRNAs
- C12N 2310/315: Phosphorothioates
- C12N 2310/321: 2'-O-R Modification
- C12N 2310/341: Gapmers, i.e. of the type ===---===
- C12N 2310/346: having a combination of backbone and sugar modifications
- C12N 2310/3515: Lipophilic moiety, e.g. cholesterol
- C12N 2310/3521: Methyl
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A01K: ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- 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)
- G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

The invention is directed to compositions and methods for treating or reducing the likelihood of the development of epilepsy in an individual. The method comprises administering to the central nervous system of an individual in need of such treatment a therapeutically effective amount of an agent capable of increasing the expression and/or activity of miR-128.

## Claims

### Claim 1

A method for treating or reducing the likelihood of the development or occurrence of seizures in an individual at risk for or having seizures, wherein said individual has suffered a prior brain injury caused by hypoxia or a prior traumatic brain injury comprising administering to said individual a therapeutically effective amount of a miR-128 having the nucleotide sequence:
(SEQ ID NO: 1)
UCACAGUGAACCGGUCUCUUU.

### Claim 2 (depends on 1)

The method of claim 1, wherein the agent is administered intrathecally or intranasally.

### Claim 3 (depends on 1)

The method of claim 1, wherein the agent is administered to the central nervous system of the individual.

### Claim 4 (depends on 1)

The method of claim 1, wherein the agent is administered to the hippocampus or cortex.

### Claim 5 (depends on 1)

The method of claim 1, wherein the brain injury is caused by is hypoxia.

### Claim 6 (depends on 5)

The method of claim 5, wherein the hypoxia is caused by stroke.

### Claim 7 (depends on 5)

The method of claim 5, wherein the agent is administered intrathecally or intranasally.

### Claim 8 (depends on 1)

The method of claim 1, wherein said individual has suffered a prior traumatic brain injury.

### Claim 9 (depends on 8)

The method of claim 8, wherein the agent is administered intrathecally or intranasally.